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Technology Name
Briefcase
Scientist
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
1517
Psychological disorders (e.g. schizophrenia, depression) are among the most prevalent diseases of humankind. These disorders affect approximately 16% of the U.S. population aged 18 and older in a given year, and when less severe conditions are considered as well (e.g. obsessive-compulsive behavior),...

Psychological disorders (e.g. schizophrenia, depression) are among the most prevalent diseases of humankind. These disorders affect approximately 16% of the U.S. population aged 18 and older in a given year, and when less severe conditions are considered as well (e.g. obsessive-compulsive behavior), the percentage is even higher (about 26%). The conventional treatments for such disorders are psychotherapy and nontherapeutic medications. Using these medications however is complicated by side effects and limitations in the amount of time they can be administered.  So far, no disease-modifying therapy has been available for any of these disorders

The present technology, developed by Prof. Michal Schwartz and her team, offers the use of molecules related to a process called sensorimotor gating which is impaired in various psychological disorders. These molecules, such as modified Kisspeptins, can reverse alterations in sensorimotor gating and provide a potential therapeutic to these hard-to-treat conditions.

Applications


•                     Potentially treating various psychological disorders – such as schizophrenia, depression, post-traumatic stress disorder (PTSD), attention deficit disorder, and others.

•                     Possible preventative treatment for the abovementioned disorders.

•                     Novel alternative approach for treating diseases that do not respond well to presently available pharmaceutical agents.


Advantages


 


Technology's Essence


The research group of Prof. Schwartz studied the connection between the immune system and neurological diseases, particularly those that manifest during adolescence. One of the parameters that characterize many such brain disorders is sensorimotor gating, the ability to segregate specific stimuli from the background of constant sensory information. Sensorimotor gating can be measured by a process called PPI (pre-pulse inhibition). PPI is also impaired in immunocompromised mice models, such as immune deficient SCID mice and a model used for schizophrenia studies. The group showed that kisspeptin, the ligand of GPR54 which regulates pubertal development, is involved in the regulation of the sensorimotor gating in vivo. Injection of kisspeptin to SCID mice reversed the abnormal PPI pattern in adults, and can therefore serve as a potential therapy for diseases associated with distorted sensorimotor gating.

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  • Prof. Michal Eisenbach-Schwartz
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
1642
A potential target for anticancer drug design.Cancer is the second leading cause of death in the US, accounting for roughly 23% of all deaths (as of 2008), and with estimated cost of care of $157 billion (as of 2010). Despite major advances in the management of cancer, most types of solid tumors remain...

A potential target for anticancer drug design.
Cancer is the second leading cause of death in the US, accounting for roughly 23% of all deaths (as of 2008), and with estimated cost of care of $157 billion (as of 2010). Despite major advances in the management of cancer, most types of solid tumors remain resistant to conventional treatment modalities. The local microenvironment, or niche, of a cancer cell plays important roles in cancer development. A major component of the niche is the extracellular matrix (ECM), a rich mesh of fibrous proteins surrounding cells that has been shown to exert a protective and supporting effect on cancer progression. Therefore, targeting the ECM represents a novel avenue for rational anticancer drug design. The current technology allows for specific targeting of an enzyme that takes part in ECM assembly and maintenance, and may provide a novel means for combating cancer progression and metastasis emergence.

Applications


  • Inhibitory antibodies to block QSOX1 catalytic activity in ECM as means to combat cancer progression and metastatic disease.
  • ]QSOX1 inhibition may also be utilized to treat lamin-associated disease.

Advantages


  • Since QSOX1 functions outside cells it would be accessible to antibodies that are not readily taken up by cells.
  • Since the microenvironment confers anticancer therapy resistance, a treatment that specifically targets the stromal cells may be synergistically combined with existing therapeutic modalities.

Technology's Essence


A team of researchers at the Weizmann Institute have that a secreted disulfide bond catalyst known as Quiescin sulfhydryl oxidase 1 (QSOX1) is required for proper assembly of the ECM. The main substrates of QSOX1 within the ECM are laminins. Thus, cells lacking QSOX1 show poor incorporation of laminin into the ECM mesh, resulting in decreased cell adherence and perturbed cell migration. Notably, in some cancer types such as pancreatic and breast cancers, QSOX1 and the ECM components it produces are over-expressed. This suggests that modulation of QSOX1 activity may provide a novel means to combating cancer and metastasis. Treatment of cancer cells with a monoclonal QSOX1-targeting antibody effectively blocked cell migration and provides a novel strategy for cancer treatment by QSOX1 inhibition.

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  • Prof. Deborah Fass

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