Gene therapies continue to advance in preclinical and clinical studies, offering hope for those in need and working to overcome the numerous drawbacks and side effects of traditional viral gene therapy approaches.
Current viral-mediated gene transfer research for treatment of Alzheimer’s disease
Oct 18, 2018Alzheimer’s disease (AD) is the most common form of dementia and has affected millions of individuals worldwide. The hallmarks of AD include the amyloid beta plaque deposits, tau neurofibrillary tangles, altered neuronal signaling, alongside decline in memory...
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Promise of gene therapy to treat sickle cell disease
Oct 16, 2018Sickle cell anemia (SCA) is a hereditary blood disease caused by a single-gene mutation that affects millions of individuals world-wide. In this review, we focus on techniques to treat SCA by ex vivo genetic manipulation of hematopoietic stem/progenitor cells (HSPCs)...
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Optimized Subretinal Injection Technique for Gene Therapy Approaches
Oct 16, 2018Gene therapy for inherited eye diseases requires local viral vector delivery by intraocular injection. Since large animal models are lacking for most of these diseases, genetically modified mouse models are commonly used in preclinical proof-of-concept studies.
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