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Technology Name
Briefcase
Scientist
1650
Efficient Production of natural Astaxanthin in bioengineered bacteria is a game changer for the nutraceuticals industry. The market-pull for natural Astaxanthin is much greater than the supply. Synthetic Astaxanthin is produced from petrochemical sources; it contains unwanted stereoisomers and is...

Efficient Production of natural Astaxanthin in bioengineered bacteria is a game changer for the nutraceuticals industry. The market-pull for natural Astaxanthin is much greater than the supply. Synthetic Astaxanthin is produced from petrochemical sources; it contains unwanted stereoisomers and is rejected by consumers who prefer natural Astaxanthin. Production of natural Astaxanthin in microalgae is laborious, expensive, and time-consuming.
Researchers at the Weizmann Institute used a combinatorial approach to construct bioengineered operons capable of modulating the expression levels of enzymes involved in the production of Astaxanthin. By combinatorial pairing of these genes in E. coli, they achieved natural Astaxanthin production 4-fold higher than previously reported.
The innovative method can challenge the deficiencies of natural Astaxanthin production in microalgae. Following scale-up and industrial development of the proprietary process, production of natural Astaxanthin has the potential to be considerably cheaper and competitive with the cost of synthesizing Astaxanthin.

Applications


  • Cost-effective Production of natural Astaxanthin for the nutraceuticals industry, animal feed industry, and others.
  • A doorway to the generation of many future products in E. coli, specifically metabolites that are produced in elaborate metabolic pathways.

Advantages


  • Full control over carotenoid accumulation profile.
  • Cheaper, straightforward generation of Astaxanthin in E. coli as opposed to generation in algae which involves high raw materials cost, land usage, air emissions etc.
  • Natural Astaxanthin as opposed to synthetic, uncontaminated with intermediate compounds and stereoisomers.

Technology's Essence


At Dr. Ron Milo’s lab researchers employed a method that uses the relatively short Ribosome Binding Site (RBS) sequence in a combinatorial manner. The methodology involves combinatorial pairing of target genes (Astaxanthin metabolic pathway enzymes) with a small set of RBS sequences and assembling them into a library of synthetic operons to explore protein expression space and to locate desired phenotypes in bacteria.
The researchers used a small set of RBS sequences to modulate in parallel the protein expression levels of multiple genes over several orders of magnitude. Using this approach, they were able to efficiently scan a large fraction of the Astaxanthin metabolic expression space with a manageable set of tested genotypes.

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  • Prof. Ron Milo
1610
A novel method for increasing Insulin content in pancreatic beta cells. In healthy individuals, Insulin is produced by beta cells of the pancreas. In people with type 1 diabetes mellitus (T1DM), these cells do not produce enough Insulin to effectively fine-tune blood sugar levels. In the US alone...

A novel method for increasing Insulin content in pancreatic beta cells.

In healthy individuals, Insulin is produced by beta cells of the pancreas. In people with type 1 diabetes mellitus (T1DM), these cells do not produce enough Insulin to effectively fine-tune blood sugar levels. In the US alone there are up to 3 million affected individuals with 30,000 new cases diagnosed each year. Worldwide, T1DM incidence has been increasing in recent years by 2% to 5%. Traditionally treated by multiple daily injections of recombinant Insulin, T1DM management represents a significant burden to both patients and the healthcare system. Recent data estimate that T1DM costs the US ~$15 billion annually in medical costs and lost income. Thus, novel therapeutic approaches to amplify Insulin production in diseased beta cells or to replace them entirely are in great need. The present technology describes a cell-based method to enhance beta cell differentiation and Insulin production by the downregulation of a pancreas-enriched microRNA.

 

Applications


  • Cell replacement therapy: directed differentiation of stem cells towards a beta cell fate followed by transplantation of these engineered cells into patients.
  • These methods can potentially be applied to other Insulin deficiency-related conditions such as diabetes mellitus type 2, metabolic syndrome and obesity.

Advantages


  • Simple and robust method for accelerating beta cell differentiation.
  • Cell base therapy for diabetes.
  • Increasing Insulin level.

Technology's Essence


A research team headed by Dr. Hornstein from the Weizmann Institute has discovered an essential role for microRNA-7 (miR-7), a microRNA that is highly and selectively expressed in the endocrine pancreas, in the regulation of beta cell differentiation. By down-regulating the expression of miR-7, the researchers were able to accelerate beta cell differentiation, and concomitantly to augment their Insulin production rate. The data gained from these studies can be further utilized in cell-based therapy applications to restore Insulin production in damaged beta cells, or alternately to replace these cells with stem cells coaxed to differentiate towards a beta cell fate.

 

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  • Dr. Eran Hornstein
1662
Immunotherapy, that is the use of the immune system to treat cancer, is currently a leading candidate in the combat against cancer. Unlike the toxic effects of both chemotherapy and radiation, immunotherapy is considered to have mild side effects due to its ability to differentiate between healthy and...

Immunotherapy, that is the use of the immune system to treat cancer, is currently a leading candidate in the combat against cancer. Unlike the toxic effects of both chemotherapy and radiation, immunotherapy is considered to have mild side effects due to its ability to differentiate between healthy and cancerous cells. Also, the therapeutic role of the immune system is an essential element in the healing process due to bone marrow transplantation for hematologic malignancies.
However, a more efficacious and less toxic T cells based treatment is required. Effective therapy depends on the functional avidity between T cell receptors (TCRs) and peptide-MHC complex (pMHC). However the natural affinity of TCR is low and they do not naturally undergo the processes that improve antibody affinity, such as somatic hypermutation (SHM). Currently there is no method of increasing the affinity of a TCR to its ligand. Moreover there is no knowledge on how use affinity maturated TCRs for creating anti-tumor reactive cells
This technology presents a method of increasing the affinity of a TCR to its ligand. This is done by subjecting TCR genes to SHM via the enzyme Activation Induced cytidine Deaminase (AID). The technology further provides affinity maturated TCRs (in cell- bound or in soluble form) and their pharmaceutical potential for immunotherapy. 

Applications


  • Generating anti-tumor T cells
  • Generating T cells reactive against selected antigen

Advantages


  • Rapid
  • Effective

Technology's Essence


This novel technology reveals that the affinity of a TCR to its ligand may be increased remarkably by subjecting TCR genes to SHM, directed by AID.
First a nucleic acid construct encoding a TCR gene is expressed in a host cell. Next SHM is used to introduce mutations to the TCR gene. Last, the the cells will be analyzed for affinity maturation by tetramer staining and subsequently sorted by FACS.
There are three parallel approaches to perform affinity maturation for the TCR: (1) Ex-vivo affinity maturation system, using Tet-regulated expression of AID (2) Ex-vivo affinity maturation system, using controlled expression of AID by mRNA electrophoresis (3) In-vitro affinity maturation system, using extracts from cells that are in SHM and recombinant AID.

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  • Prof. Rachel Lea Eisenbach
1621
Novel treatment for angiogenesis-related diseases.Angiogenesis — the growth of new blood vessels from pre-existing vasculature — has an essential role in development, reproduction and repair. Pathological angiogenesis is a common theme in a broad range of diseases such as cancer, autoimmune diseases,...

Novel treatment for angiogenesis-related diseases.Angiogenesis — the growth of new blood vessels from pre-existing vasculature — has an essential role in development, reproduction and repair. Pathological angiogenesis is a common theme in a broad range of diseases such as cancer, autoimmune diseases, age-related macular degeneration and atherosclerosis. The global market for angiogenesis stimulators and inhibitors is forecast to reach ~US $50 billion by the year 2015. Most of the currently marketed angiogenesis regulators, such as Avastin, typically display modest efficacy and therefore further highlight the great need for the development of novel therapeutics. The current technology presents a novel method to treat angiogenesis-related disorders by modulating apolipoprotein B (ApoB).

Applications


  • ApoB is a potential therapeutic target for the treatment of cancer and other non-neoplastic diseases.
  • ApoB levels may serve as a biomarker for cancer metastasis.

Advantages


  • The anti-angiogenic effect of LDL administration was demonstrated in vivo, in zebrafish models, as well as in vitro, in relevant human cells lines.
  • Regulation of ApoB levels may be applied to treat a broad range of angiogenesis-dependent diseases.
  • Detection of ApoB levels can be readily achieved by analysis of body fluids such as blood and plasma.

Technology's Essence


Using a high-throughput genetic screen for vascular defects in zebrafish, researchers at the Weizmann Institute of Science have identified a genetic mutation that leads to excessive angiogenesis. The mutated gene is responsible for the assembly of ApoB-containing lipoproteins such as LDL, otherwise known as the ‘bad’ cholesterol. The group has found that low levels of LDL promote the formation of new blood vessels by directly interacting with the VEGF pathway. The outlined technology offers methods to modulate the levels of ApoB in order to stimulate, or inhibit angiogenesis, dependent on the therapeutic strategy. For example, inhibition of angiogenesis by increasing ApoB levels may repress tumor growth and attenuate its metastatic potential. In another application of this technology, increased circulating levels of ApoB can serve as a biomarker for the overproduction of blood vessels, thus enabling early diagnosis of pathogenic states in angiogenesis-dependent diseases.

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  • Prof. Karina Yaniv
1518
Improved immunotherapy for breast cancer. Monoclonal antibodies (mAbs) to ErbB-2/HER2 growth factor receptor, or to its sibling, the epidermal growth factor receptor (EGFR), prolong survival of cancer patients, especially when combined with cytotoxic therapies. However, low effectiveness of...

Improved immunotherapy for breast cancer.

Monoclonal antibodies (mAbs) to ErbB-2/HER2 growth factor receptor, or to its sibling, the epidermal growth factor receptor (EGFR), prolong survival of cancer patients, especially when combined with cytotoxic therapies. However, low effectiveness of therapeutic mAbs and the evolution of patient resistance call for improvements. Furthermore, the response to the clinically approved monotherapy of Herceptin (a humanized mAb directed against ErbB-2), is relatively low (~15%) and short lived (median duration, 9 months). Therefore, there is a need to improve the therapeutic treatment against this receptor. The present technology enhances the therapeutic activity of anti-ErB-2 receptor antibodies, by combining two or more epitope-distinct antibodies.

Applications


  • Improved treatment of ErbB-2-overexpressing tumors (e.g. in breast and ovary cancers).


Advantages


  • May enhance patient response and delay acquisition of resistance.
  • Enhancement of therapeutic efficacy and synergy with chemotherapy.

Technology's Essence


Optimal selection of mAbs for cancer immunotherapy may improve its therapeutic potential. The outlined technology addresses an emerging strategy, which enhances the therapeutic activity of anti-receptor antibodies by combining two mAbs engaging distinct epitopes. It was demonstrated that pairs of anti-ErbB-2 mAbs better inhibit ErbB-2-overexpressing tumors than the respective individual mAbs, both in vitro and in vivo.

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  • Prof. Yosef Yarden
1033
A novel diagnostic test to identify individuals with increased risk of lung cancer. Lung cancer is the number one killer among cancers, with 160,000 deaths/year in the USA and 1.6 million/year worldwide. Early detection of lung cancer increases 5-year survival rate from 4% to 54%. Moreover, the...

A novel diagnostic test to identify individuals with increased risk of lung cancer.

Lung cancer is the number one killer among cancers, with 160,000 deaths/year in the USA and 1.6 million/year worldwide. Early detection of lung cancer increases 5-year survival rate from 4% to 54%. Moreover, the National Lung Cancer Trial (NLST) showed that early detection of lung cancer by low-dose CT reduces mortality by at least 20%. Despite recommendations for low-dose CT screening for heavy smokers fulfilling the NLST criteria, compliance is low. In addition, 80 million smokers and ex-smokers in the US who do not fulfil NLST risk criteria have no recommended solution.

The MyRepair test fulfils this unmet medical need by providing a quantitative prediction of lung cancer risk using a simple blood test. The test is based on a personalized measurement of the patient’s DNA repair capacity, a mechanism which is highly connected to the onset of cancer. Therefore, the MyRepair technology can potentially increase early detection of lung cancer and thus save lives.

 

Applications


A novel diagnostic test to identify individuals with increased risk of lung cancer


Advantages


·         Simplicity – MyRepair is based on a simple, cost-effective blood test.

·         Accessibility – Compared to low-dose CT which requires specific equipment, the MyRepair test can be easily integrated in general diagnostic labs and therefore may be more accessible to a larger portion of the population.

·         Additional applications – Since the test is based on measuring personalized DNA repair mechanism, it can be adopted in the future for the diagnosis of additional cancer types and DNA repair related diseases.


Technology's Essence


Based on the strong and well documented connection between impaired capacity for DNA repair and onset of cancer, the Livneh lab invented the MyRepair Test, a method for predicting lung cancer risk, based on measuring activity of 3 DNA repair enzymes.

Combining enzyme activities with experimental risk estimates generated MyRepair Score, which measures personalized DNA repair capacity of tested subjects.

An epidemiological/clinical study performed in Israel, further validated in an independent UK study, demonstrated that lung cancer patients have lower MyRepair Score than healthy people. In addition, subjects who test MyRepair-positive have an 85-fold higher risk to develop lung cancer compared to the general population.

Low MyRepair Score is a risk factor independent of smoking, and of comparable magnitude, indicating that it can be a prognostic tool for smokers, ex-smokers, and non-smokers.

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  • Prof. Zvi Livneh
1270
Monoclonal antibodies to IgE Description: Rat monoclonal anti-IgE antibodies that was generated by fusion of plasmacytoma (84.1C) or myeloma (EM953) cells with splenocytes of rat immunized with purified murine IgE mAb. The antibodies react with various IgE mAb of different specificities and not with...

Monoclonal antibodies to IgE

Description: Rat monoclonal anti-IgE antibodies that was generated by fusion of plasmacytoma (84.1C) or myeloma (EM953) cells with splenocytes of rat immunized with purified murine IgE mAb. The antibodies react with various IgE mAb of different specificities and not with immunoglobulins of other classes, and recognize an epitope on the murine Fc epsilon region.

Were shown to block IgE-Fc?R interactions and inhibit passive cutaneous anaphylaxis. 

Clone 84.1c recognizes a site on IgE, which is identical or very close to the Fc?R binding site. May be used for detection and manipulation of the IgE response in mice.

Reference:  Schwarzbaum S, Nissim A, Alkalay I, Ghozi MC, Schindler DG, Bergman Y, Eshhar Z. 1989. Mapping of murine IgE epitopes involved in IgE-Fc epsilon receptor interactions. Eur J Immunol 19(6):1015-23.

 

M182, M185, M186

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  • Prof. Zelig Eshhar
1397
A novel antibody which can be used, for the first time, to recognize ubiquitinated histone 2B. This technology is novel in its ability to recognize proteins and their destinations, and may serve in diagnostics and immunoprecipitation processes.

A novel antibody which can be used, for the first time, to recognize ubiquitinated histone 2B. This technology is novel in its ability to recognize proteins and their destinations, and may serve in diagnostics and immunoprecipitation processes.

Applications


Primary applications in research. Use as a detection tool in western blotting, immunoprecipitation and chromatin immunoprecipitation. Might be used for monitoring processes associated with modulations of ubiquitinated-H2B levels.

Technology's Essence


The invention involves the generation of antibodies specific to ubiquitinated-H2B which selectively recognize H2B when it is ubiquitinated but not H2B in its unmodified state, or ubiquitin unconjugated to H2B.

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  • Prof. Moshe Oren
1499
Bladder cancer is a common malignancy; it is the 4th most common cancer in males and the 9th in females.  The presenting symptom is usually blood in the urine, and diagnosis is currently based on cystoscopy, which is invasive, costly, painful and time consuming.  To date, no biomarker has been...

Bladder cancer is a common malignancy; it is the 4th most common cancer in males and the 9th in females.  The presenting symptom is usually blood in the urine, and diagnosis is currently based on cystoscopy, which is invasive, costly, painful and time consuming.  To date, no biomarker has been identified in the urine that might be used for screening, staging, prognosis and monitoring treatment.  We now report that the amount of the 60 kDa heat shock protein (HSP60) in a subject’s urine is a biomarker for muscle invasion in patients with bladder cancer – stage T2 and higher.  Moreover, subjects with stage T1 disease can be stratified by their urine levels of HSP60 into a sub-group likely to progress into stage T2 or into a sub-group more likely to respond to conservative treatment with BCG, which does not require removal of the bladder.  The distinction between these two sub-groups of T1 bladder cancer can identify earlier subjects in need of cystectomy, while sparing others unnecessary major surgery.

Applications


  • Screening subjects with overt hematuria, or at risk of developing bladder cancer (such as heavy smokers)
  • tratifying bladder cancer subjects
  • Prognosis
  • Determining treatment program
  • Monitoring response to therapy.

Advantages


  • Non-invasive
  • Easy to apply
  • Relatively inexpensive
  • Prognositic.

Technology's Essence


Quantitative measurement of HSP60 levels in a subject’s urine by ELISA, radio-immunoassay or other simple assays.

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  • Prof. Irun R. Cohen
1245

Applications


The novel DNA Aptamer is a promising candidate for therapeutic as well as diagnostic uses: Therapeutic: A novel therapy for Influenza Diagnostics: Detection of Influenza infection in vertebrates such as avian, swine and human

Technology's Essence


Scientists at the Weizmann Institute of Science describe a novel oligonucleotide, also known as an Aptamer, which has been designed to complement the receptor-binding region of the influenza haemagglutinin molecule. It was constructed by screening a DNA library and processing by the SELEX procedure. This DNA Aptamer comprises of a polynucleotide sequence that can bind to a polypeptide within the binding region of the influenza virus to the host cell. The proposed mode of action of this Aptamer is by blocking the binding of influenza virus to target cell receptors and consequently preventing the virus invasion into the host cells. Aptamer is capable of inhibiting the haemagglutinin capacity of the virus and the viral infectivity in vitro. Furthermore, it was shown in an animal model to inhibit viral infection by different influenza strains, as manifested by up to 99% reduction of virus burden in the lungs of treated mice.

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  • Prof. Ruth Arnon
1369
A simple, single-step biochip platform for synthesis of biomolecules. Biochip technology is used today in measuring passive probe-target interactions i.e. measurement of the abundance of specific biomolecules). This technology can now be extended to include complex and cascaded activities on the chip...

A simple, single-step biochip platform for synthesis of biomolecules.

Biochip technology is used today in measuring passive probe-target interactions i.e. measurement of the abundance of specific biomolecules). This technology can now be extended to include complex and cascaded activities on the chip. The present immobilization approaches (based on UV photography) have been essentially limited to short single stranded DNA probes and have not been developed for entire genes or other biochemical functions. Furthermore, most biochips are assembled in a multi-step process that requires expertise in surface chemistry in order to obtain reproducibility and robustness. As a result, light-directed immobilization of molecules on biochips is not widespread and is not easily accessible for research and technology development. The present invention enables, in a simple manner, to immobilize different biomolecules anywhere on the chip to submicron resolution through selective exposure of the monolayer to UV light.

 

Applications


  • Light-directed immobilization of a variety of different biomolecules (e.g. DNA, antibodies, enzymes and peptides)
  • On-chip protein biosynthesis from immobilized genes
  • Design and layout of on-chip traps for proteins from crude cell extract
  • Lab-on-a-chip that provides a general use biochip technology

Advantages


  • Enabling the use of long DNA molecules (whole genes)
  • Robust and simple performance without the need for proficiency in materials science and surface chemistry
  • On-chip protein synthesis with high efficiency, minimal non-specific activity, and a wide dynamic range

 


Technology's Essence


This lab-on-a-chip technology (i.e. a technology that enables to perform laboratory operations on a small scale) is based on a newly synthesized molecule termed daisy that combines three parts all-in-one: a tail and head connected by a backbone. Selective exposure of daisy monolayer to UV light through a mask (photolithography) reveals the surface for chemical binding of a variety of biomolecules. Using this technology it is possible to immobilize different biomolecules anywhere on the chip to submicron resolution. By immobilizing whole genes, thus enabling cell-free biosynthesis of proteins, daisy technology takes the lab-on-a-chip concept to the next level. Daisy biochip technology holds a promise in proteomics, diagnostics and therapeutics.

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  • Prof. Roy Bar-Ziv
1441
New protein as a target to treat B cell-related cancer.Chronic lymphocytic leukemia (CLL), a malignant disease characterized by the accumulation of B lymphocytes in the blood, lymphoid organs, and bone marrow, is the second most common type of leukemia in adults, accounting for about 7,000 new cases of...

New protein as a target to treat B cell-related cancer.
Chronic lymphocytic leukemia (CLL), a malignant disease characterized by the accumulation of B lymphocytes in the blood, lymphoid organs, and bone marrow, is the second most common type of leukemia in adults, accounting for about 7,000 new cases of leukemia each year. Presently, there is no cure for CLL, and the overall goal of leukemia treatment is to bring about a remission. Therefore, identifying new proteins that may serve as a target for inducing cell death in the malignant cells is highly desirable. The present technology identifies a new regulator protein that is essential for the survival of CLL cells.

Applications


• Treatment of CLL, as well as other B cell-related cancers (e.g. gastric cancer and renal cell carcinoma), by blocking CD84 activity
• Diagnosis of CLL

Advantages


• Very specific to malignant B cells
• Diagnosis, and therefore treatment, can be made at early stages of the disease

 


Technology's Essence


B cells taken from CLL patients have a high level of the protein CD84. Stimulation of CD84 upregulates the survival of B-CLL. However, inhibition of CD84 activity with a blocking antibody downregulates the expression of another protein which controls B-CLL survival, thus inducing cell death. Therefore, the present invention reveals CD84 as a regulator of B-CLL survival

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  • Prof. Idit Shachar
1655
Cellular senescence is a permanent cell cycle arrest induced by damage or stress applied on proliferating cells. In a cell autonomous manner, senescence is a potent barrier to tumorgenesis and contributes to the cytotoxicity of some anti-cancer drugs. However, with age senescence cells accumulate and...

Cellular senescence is a permanent cell cycle arrest induced by damage or stress applied on proliferating cells. In a cell autonomous manner, senescence is a potent barrier to tumorgenesis and contributes to the cytotoxicity of some anti-cancer drugs. However, with age senescence cells accumulate and promote a number of pathological conditions. Therefore the elimination of senescent cells is desired in order to prevent tumor- and inflammation- related pathologies and also to inhibit tissue ageing.
Today, our understanding of the mechanisms regulating the viability of senescent cells is limited. It has been suggested that senescent cells are resistant to apoptosis. Therefore, senescent cells elimination may be achieved by modifying the resistance to apoptosis of these cells.
Here the researches demonstrate the first feasible therapeutic approach that leads to eradication of senescent cells. Combination of direct induction of apoptosis in senescent cells with induction of cell death by pro-inflammatory repose induce by p21 knockdown will lead to reduction of viable senescent cells.

Applications


  • A therapeutic impact on inflammatory and fibrotic disease
  • Therapy for age-related disease such as type 2 diabetes, Alzheimer’s disease, Atherosclerosis, cataracts, Chronic obstructive pulmonary disease (COPD), and Osteoporosis

Advantages


  • Effective elimination of senescent cells- removal of senescent cells can prevent or delay tissue dysfunction and extend health span
  • Does not damage normal cells even at high concentrations

Technology's Essence


Researches demonstrated that the anti-apoptotic proteins Bcl-xL and Bcl-w level were elevated in senescence cells of both human and mouse origin. A subsequent study, in which Bcl-xL and Bcl-w were knocked down by siRNA, revealed that a combined knock down of Bcl-xL and Bcl-w had synergic effect, resulting in reduction of 50% in cell viability. Thus the increased level of anti-apoptotic proteins Bcl-xL and Bcl-w may account for the apoptotic resistance of senescent cells. p21 knockdown induced pro-inflammatory response and cell death in senescent cells.
Overall, the researchers show that combined inhibition of the anti-apoptotic proteins Bcl-xL and Bcl-w allows specific elimination of senescent cells and might be used to treat diseases where senescent cells are present. The researchers also found that the same effect might be achieved by reducing the expression of p21 in senescent cells. Integrating both approaches propose a more effective therapy.

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  • Prof. Valery Krizhanovsky
1498
MicroRNAs as potential biomarkers for ALS.Amyotrophic Lateral Sclerosis (ALS) is a devastating disease that progressively destroys motor neurons in the brain and the spinal cord, eventually causing paralysis and death. Currently, there are approximately 25,000 patients with ALS in the USA, with a...

MicroRNAs as potential biomarkers for ALS.
Amyotrophic Lateral Sclerosis (ALS) is a devastating disease that progressively destroys motor neurons in the brain and the spinal cord, eventually causing paralysis and death. Currently, there are approximately 25,000 patients with ALS in the USA, with a median age of onset of 55 years. Approximately 5–10% of patients with ALS have a family history, and these patients most frequently inherit the disease in an autosomal dominant manner. Family-based linkage studies have led to the identification of several genes for familial ALS. However these findings only explain a small fraction of all ALS cases. The majority of ALS cases have no obvious family history and are referred to as sporadic ALS. At present, there is no effective therapy for the disease and patients usually die within 2-5 years after the onset of symptoms. Thus, there is an urgent need for biomarkers that could substantially aid early diagnosis of ALS and will help in designing decisive clinical trials of new drugs. The present technology provides specific microRNAs that can serve as potential biomarkers for ALS.

Applications


  • Unique patterns of microRNA expression profile in the cerebrospinal fluid of ALS patients could be useful as molecular biomarkers for disease diagnosis and eventually prediction of therapeutic responses.
  • The suggested ALS biomarkers may be employed in drug development studies.

 


Advantages


  •  MicroRNAs can be precisely quantified using qRT-PCR that provides exceptionally high sensitivity and specificity of detection.
  • The small size of microRNAs offers a unique advantage since they are more stable and less prone to enzymatic degradation, and are therefore amenable to an accurate assessment of their expression levels.

Technology's Essence


MicroRNAs (miRNAs) are endogenous small noncoding RNAs that negatively regulate gene expression in a posttranscriptional fashion and contribute to a wide variety of biological processes. miRNAs play important roles in the development of the central nervous system and their involvement in neurodegenerative diseases such as Parkinson's disease and Alzheimer’s disease has been recently established. The outlined technology describes specific miRNAs that are enriched in motor neurons and are significantly downregulated in mouse models of hereditary motor neuron disease (SOD1G93A and SMN1). These miRNAs may serve as putative biomarkers for motor neuron diseases such as ALS by measurement of their expression levels in cerebrospinal fluid samples collected from affected individuals.

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  • Dr. Eran Hornstein
1698
GD is an inherited metabolic disorder, affecting about 1 in 20,000 births. GD is divided into three clinical subtypes: type 1 is the most common and is characterized by bruising, fatigue, anemia, low blood platelets, and enlargement of the liver and spleen. Types 2 and 3, also called neuronopathic GD (...

GD is an inherited metabolic disorder, affecting about 1 in 20,000 births. GD is divided into three clinical subtypes: type 1 is the most common and is characterized by bruising, fatigue, anemia, low blood platelets, and enlargement of the liver and spleen. Types 2 and 3, also called neuronopathic GD (nGD), affect 4% of GD patients and additionally include neurological symptoms. Type 1 patients can have a normal life expectancy if treated whereas type 2/3 patients do not survive to reach adulthood. Moreover, GD carriers, approximately 1% of the population, are in a major risk of developing Parkinson’s disease. Current therapies suffer from severe drawbacks in the treatment of type 1 GD and no therapy exists that effectively treat nGD. The present technology offers a novel therapeutic target for the treatment of Gaucher's disease (GD) which addresses also the neurological symptoms.

Applications


  • Alternative treatment for type 1 GD
  • First line therapy for nGD

Advantages


  • A novel therapy for nGD which has no treatment for the present.
  • A novel therapeutic approach for GD type 1, via a previously unknown molecular mechanism.
  • Allows the development of an orally administered treatment, far more convenient for the patients than the existing treatments.
  • Reduced costs compared to the existing therapies of ERP or BMT

Technology's Essence


The proposed technology is based on the discovery that RIP3 is a key player in the manifestation of GD and that inhibiting RIP3 activity is effectively ameliorating the symptoms of GD not only in the less severe type 1 but also in the neuropathic form of the disease, types 2 and 3. nGD is associated with a massive neuronal loss and elevated RIP3 levels. Inhibition of RIP3 in a mouse model of nGD resulted in a dramatic attenuation of disease signs: drastic extension of life span, no weight loss, improvements in motor coordination, reduced neuroinflammation and improved liver and spleen injuries.

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  • Prof. Anthony H. Futerman

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