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Showing 4 matches for the following: Inventor: "Asokan, Aravind" ×

Gene therapy for long-term treatment of GSDIa

Gene therapy for long-term treatment of GSDIa

Unmet Need Glycogen storage disease type Ia (GSDIa), or von Gierke disease, is a rare heritable metabolic disorder that impacts the liver and kidneys due to an inability to break down glycogen. An estimated 1…

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Methods and compositions for treating protein aggregation diseases

Methods and compositions for treating protein aggregation diseases

Unmet Need Neurodegenerative disease affects more than 50 million people per year. Certain neurodegenerative diseases, like Alzheimer’s disease, result from the accumulation of amyloid-forming proteins in the brain, which results in cognitive impairment and has…

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Gene therapy for ATP1A3-Related Neurologic Disorders

Gene therapy for ATP1A3-Related Neurologic Disorders

Unmet Need Children with Alternating Hemiplegia of Childhood can have episodes of temporary paralysis, dystonia, and severe behavioral problems. The prevalence of AHC is estimated to be 1 in one million to 1 out of…

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Method to improve Adeno-Associated Virus (AAV) gene delivery by evading breakdown by the immune system

Method to improve Adeno-Associated Virus (AAV) gene delivery by evading breakdown by the immune system

Unmet Need Adeno-Associated Virus (AAV) vectors are a safe and effective way to deliver gene therapies to patients. However, one limitation of AAV-based therapies is caused by the patient’s immune system. About 30-60% of patients…

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