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Shilpa Prabhakar on Muck Rack

Shilpa Prabhakar

As seen in: Nature, Science Magazine
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Articles

Engineered AAV capsids mediate transduction of murine neurofibroma and sciatic nerve - Gene Therapy

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By Shilpa Prabhakar, Nate Jowett, Xandra O. Breakefield, Edwina Abou Haidar
| Nature Verified
Abstract Genetic diseases such as Neurofibromatosis type 1 (NF1) and Charcot-Marie Tooth disease involve Schwann cells (SCs) associated with peripheral nerves. Gene therapy using adeno-associated virus (AAV) vector mediated gene delivery is a promising strategy to treat these diseases. However, AAV-mediated transduction of SCs in vivo after intravascular delivery is relatively inefficient, with a lack of extensive characterization of different capsids to date.
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Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin

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By David Yellen, Roberta L. Beauchamp, Pike-See Cheah, Shilpa Prabhakar
| Science Magazine Verified
Abstract Tuberous sclerosis complex (TSC) results from loss of a tumor suppressor gene - TSC1 or TSC2, encoding hamartin and tuberin, respectively. These proteins formed a complex to inhibit mTORC1-mediated cell growth and proliferation. Loss of either protein leads to overgrowth lesions in many vital organs. Gene therapy was evaluated in a mouse model of TSC2 using an adeno-associated virus (AAV) vector carrying the complementary for a “condensed” form of human tuberin (cTuberin).
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