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Therapeutic-viral mediated gene delivery in the 21st century

Friday, November 08, 2013 — Concurrent Symposia Session IV

12:00 p.m. – 2:00 p.m.

FAES Academic Center lower-level Classrooms 1 and 2


  • Maribeth Eiden, NIMH


The continuing discovery of the genetic basis of many human disorders, development of corresponding animal models, and continued advances in vector-mediated gene delivery have all increased the potential for viral vector mediated gene delivery to ameliorate human disease. Viral vector mediated gene therapy is a reiterative process requiring continual feedback from clinical trial results to ensure the proper direction of translational studies. Modifications to the design of retroviral vectors and delivery modalities based on this feedback are a path towards arresting the progression or relieving the symptoms in human diseases. This symposium will serve to put forward current findings that demonstrate what we have learned from clinical trials, in terms of biosafety considerations, targeted gene delivery and stem cell biology that are now being incorporated into the design and implementation of viral vectors.

Brain-directed viral gene therapy for neurometabolic disorders
Stephen Kaler, NICHD

Increased occurrence of hepatocellular carcinoma following adeno-associate virus (AAV) gene delivery to neonatal mice
Randy Chandler, NHGRI

Gene therapy for adenosine deaminase deficiency
Fabio Candotti, NHGRI

Reprogramming T Lymphocytes by g-retroviral vectors in adoptive immunotherapy for cancer
Steven Feldman, NCI

Innovations in the development of specialized vector design based on novel retroviral isolates
Wenqin Xu, NIMH

Overcoming antigenic diversity in the design of novel norovirus vaccines; FARE Award Winner
Gabriel Parra, NIAID

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