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3. A humanized mouse model for adeno-associated viral gene therapy

4. An evolved AAV variant enables efficient genetic engineering of murine T cells

12. The NIH Somatic Cell Genome Editing program

16. Physical positioning markedly enhances brain transduction after intrathecal AAV9 infusion

18. Evaluation of Aav Capsids and Delivery Approaches for Hereditary Hemorrhagic Telangiectasia Gene Therapy

19. Deep Learning Resolves Myovascular Dynamics in the Failing Human Heart

21. Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector

22. Transgenic mice for in vivo epigenome editing with CRISPR-based systems

25. Repurposing CRISPR-Cas13 systems for robust mRNA trans-splicing

28. An siRNA Screen Identifies the U2 snRNP Spliceosome as a Host Restriction Factor for Recombinant Adeno-associated Viruses.

30. Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.

35. AAV Gene Augmentation of Truncated Complement Factor H Differentially Rescues Ocular Complement Dysregulation in a Mouse Model

38. AAV9-mediated gene delivery to liver grafts during static cold storage in a rat liver transplant model

41. Rescue of glutaric aciduria type I in mice by liver-directed therapies

44. RESCUE OF GLUTARIC ACIDURIA TYPE I MICE BY LIVER DIRECTED THERAPIES

45. An enhancer-based gene-therapy strategy for spatiotemporal control of cargoes during tissue repair

49. Adeno-associated virus mediates gene transduction after static cold storage treatment in rodent lung transplantation

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