Search Results - Therapy+Targets

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First in class use of the estrogen receptor-b agonist in treating Angelman syndrome and relevant autism spectrum disorders

Overview Angelman syndrome is a genetic disorder caused by UBE3A gene dysfunction, leading to cognitive impairment, motor coordination issues, and developmental delays. Research suggests that oligodendroglial dysfunction, which affects myelination in the brain, contributes to these conditions. Based on the research, this invention discloses a novel...

Published: 3/6/2025 | Inventor(s): Yu-Wen Huang, Xin Yang

Keywords(s):

Category(s): Genetic Diseases, Therapeutics, Therapy Targets

Treating Glioblastoma, Metastatic Melanoma, and Other Tumors Involving CNS Metastases

Overview Metastasis involving the spread of systemic cancer to the brain typically results in neurologic disability and death; current treatments are largely palliative in nature. We have invented a method of treatment based on determining whether the patient has an increased level of MDM2/MDM4 genes and/or proteins and/or a decreased level of CDKN2A...

Published: 6/7/2023 | Inventor(s): Wafik El-Deiry, Taylor Arnoff

Keywords(s):

Category(s): Diagnostics, Therapy Targets, Oncology

HDAC-7 as novel therapeutic target for human glioblastoma

Overview Glioblastoma is one of the most aggressive and lethal human tumors. We have identified histone deacetylase 7 (HDAC-7) as a potential therapeutic target, a crucial first step toward development of an effective treatment for glioblastoma. Market Opportunity Glioblastoma is the most malignant and aggressive primary brain tumor, with a survival...

Published: 10/15/2022 | Inventor(s): Nikolaos (Nikos) Tapinos, Ola Hassan, David Karambizi

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Category(s): Therapy Targets, Oncology

Metabolite Treatments for Metabolic Dysfunction and Neurological Disease Caused by Mutations in Mitochondrial Enzyme GPT2

Overview The mitochondrial enzyme glutamate pyruvate transaminase 2 (GPT2) is necessary for metabolic mechanisms central to neuronal survival and axonal growth. Having demonstrated that loss-of-function mutations in GPT2 cause neuronal vulnerabilities involved in a variety of brain diseases and areas of brain function, we have developed a metabolite...

Published: 10/12/2022 | Inventor(s): Eric Morrow

Keywords(s):

Category(s): Metabolic diseases, Therapy Targets

Proprietary targeting of Oligonucleotide to vulnerable splicing events to efficiently silence genes

Overview The principle of antisense technology is the sequence-specific binding of an antisense oligonucleotide (ASO) to target mRNA and prevent gene translation. We have found the individual events that are most vulnerable to disruption in cancer cells and developed a method to target these events. We can further restrict the points of vulnerability...

Published: 10/12/2022 | Inventor(s): William Fairbrother, Jeremiah Buerer, David Glidden

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Category(s): Drug Discovery Platform, Therapy Targets

A novel nucleocytoplasmic regulator of autophagy-associated transcription factors stimulates autophagy and prevents the proteostatic decline associated with neurodegeneration

Activating Autophagy to Prevent Neurodegeneration and Extend Lifespan Overview Decline in proteostasis is a hallmark of age-related neurodegerative disease such as Alzheimer’s Disease. Autophagy is the process by which cells rid themselves of damaged or otherwise unnecessary material, which then moves to the lysosome and degrades. Pharmacologic...

Published: 2/22/2022 | Inventor(s): Louis Lapierre

Keywords(s):

Category(s): Therapy Targets, Neurodegenerative Disease