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Technology Name
Briefcase
Scientist
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
1446
Peptide sequences for efficient inhibition of nuclear translocation of proteins. The ability to regulate cellular localization of a biological component is important for many functions such as gene therapy, protection from toxic chemicals, transport of anti-cancer agents, and possibly preventing...

Peptide sequences for efficient inhibition of nuclear translocation of proteins.

The ability to regulate cellular localization of a biological component is important for many functions such as gene therapy, protection from toxic chemicals, transport of anti-cancer agents, and possibly preventing nuclear translocation of oncogenes. To ensure accurate cellular functioning, the spatial distribution of proteins needs to be delicately regulated and coordinated. This is particularly apparent in many signaling proteins that dynamically and rapidly change their localization upon extracellular stimulation. The present invention provides peptides that may be used to regulate the nuclear translocation of proteins that endogenously comprise such nuclear translocation signals.

Applications


  • Inhibition of translocation of endogenous oncogenes and thereby the transcription they induce.

Advantages


  • Regulation of the level of nuclear targeting activity by selection of different amino acids in the peptide sequences.

  • Peptides can be modified in order to make them more stable in the body.
  • Modulation of the nuclear activities of proteins without harming their cytoplasmic activities.

Technology's Essence


The current invention identifies a 3-amino acid domain (Ser-Pro-Ser, SPS), which is a nuclear translocation signal present in signaling proteins such as extracellular signal-regulated kinase (ERK2) protein, SMAD3 and mitogen-activated protein kinase 1 (MEK1). SPS participates in nuclear translocation upon extracellular stimulation. Since several of these proteins are involved in the regulation of cellular proliferation and oncogenic transformation, the SPS domain can compete with the translocation machinery and therefore prevent the translocation of the proteins into the nucleus. As was shown in animal models, inhibiting this mechanism has an advantage over other ways of inhibition as it doesn’t lead to a negative feedback loop which may enhance the production of the protein.

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  • Prof. Rony Seger

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