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Miguel Sena-Esteves and Xandra Breakefield
Treatment of many neurological diseases remains primarily palliative, and for some of these diseases there is little prospect for the development of effective pharmaceuticals in the near future. Although gene therapy has had its share of disappointment, perhaps due to an overly optimistic initial outlook, the continued development of novel gene delivery vectors and study of their basic biology, as well as the introduction of novel infusion/delivery techniques, has brought this field to the brink of realizing some of its awesome potential for treatment of many neurological diseases. Initially this course will focus on comparing the properties and effectiveness of different viral vector systems for gene delivery to the central nervous system, covering the use of adenovirus, adeno-associated virus (AAV), lentivirus, and herpes simplex virus (HSV) vectors. The recent developments in infusion techniques that can lead to wide distribution of viral vectors in the brain and other organs will be discussed. The following sessions will be focused on the challenges and progress made towards developing vectors for the understanding and treatment of specific neurological diseases, including dystonia, lysosomal storage diseases, ataxia-telangiectasia, Parkinson’s, Huntington’s, brain tumors, trauma, and amyotrophic lateral sclerosis, including the use of inhibitory RNAs for dominant neurogenetic diseases, such as spincerebellar ataxia. Each two hour session after the first one will include a 45 minute talk by an expert in experimental gene therapy for a particular neurological disease followed by one or two student presentations of relevant articles and discussion.
Proposed sessions: Proposed Speakers:
Vectors for gene deliveryto the nervous system Miguel Sena-Esteves and Xandra Breakefield, MGH
Dystonia Xandra O. Breakefield, MGH
Lysosomal Storage Diseases Miguel Sena-Esteves, MGH
Ataxia-telangiectasia Maria L. Cortes, MGH
Parkinson’s Disease Ole Isacson, McLean Hospital
Huntington’s Disease Anne Messer, Wadsworth Center, NY
Brain Tumors Manish Aghi, MGH
Trauma Michael Whalen, MGH
Amyotrophic Lateral Sclerosis Robert Brown, MGH
Use of shRNAs for treatment
of dominant neurogenetic diseases Beverly L. Davidson, Univ. of Iowa