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- Title
Gene therapy for CNS diseases - Krabbe disease.
- Authors
Rafi, Mohammad A.
- Abstract
This is a brief report of the 19th Annual Meeting of the American Society of Gene and Cell Therapy that took place from May 4th through May 7th, 2016 in Washington, DC, USA. While the meeting provided many symposiums, lectures, and scientific sessions this report mainly focuses on one of the sessions on the "Gene Therapy for central nervous system (CNS) Diseases" and specifically on the "Gene Therapy for the globoid cell leukodystrophy or Krabbe disease. Two presentations focused on this subject utilizing two animal models of this disease: mice and dog models. Different serotypes of adeno-associate viral vectors (AAV) alone or in combination with bone marrow transplantations were used in these research projects. The Meeting of the ASGCT reflected continuous growth in the fields of gene and cell therapy and brighter forecast for efficient treatment options for variety of human diseases.
- Subjects
GLOBOID cell leukodystrophy; GENE therapy; CENTRAL nervous system diseases; ADENO-associated virus; ANIMAL models in research; GENETICS
- Publication
BioImpacts, 2016, Vol 6, Issue 2, p69
- ISSN
2228-5652
- Publication type
Article
- DOI
10.15171/bi.2016.09