Genetic Neurodegenerative Conditions Resource Library
Explore examples of research, developmental milestones, and investigational treatment approaches targeting the central nervous system.
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Huntington’s Disease
Huntington’s DiseaseRead an in-depth analysis of Huntington’s disease characteristics, diagnosis and management approaches, and genetic testing options.
AAV Gene Therapy Research Animation
Gene TherapyAdeno-associated viral (AAV) vectors are designed to encapsulate genetic material for the potential treatment of genetic diseases. Explore the science behind AAV gene therapy research.
What Causes Genetic Disease?
Gene TherapyProteins carry out a variety of cellular and physiological functions in the body. See how changes in a person’s DNA can affect proteins and lead to genetic disease.
Gene Therapy Strategies
Gene TherapyLearn how gene therapies can potentially correct genetic mutations to provide functional protein.
Biological Features of a Recombinant AAV
Gene TherapyAAVs are commonly used as vectors in gene therapy. See how recombinant AAV vectors are designed and how they function in the body.
Targeted Cell AAV Delivery
Gene TherapyWatch how recombinant AAV vectors delivered into the body locate specific surface receptors of target cells.
Gene Therapy and the Immune Response
Gene TherapyAdministering AAV vectors can activate different branches of the immune system. Learn about this process and research to develop immunosuppressive techniques.
Parkinson’s Disease
Parkinson’s DiseaseRead an overview of the genetics of Parkinson disease and related genetic counseling issues, from clinical characteristics and causes, to evaluations strategy and genetic counseling, as well as information about targeted therapeutic trials.
Supplemental Resources
Explore additional genetic neurodegenerative conditions resources to supplement conversations with your patients.
Annual Neurology Events
At Spark Therapeutics, we’re striving to address unmet needs in genetic diseases. Here are some events relevant to our research.
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