Research >> Gene Transfer

Adeno-Associated Virus Mediated Gene Therapy

The collective experience with Adeno-associated virus (AAV) vector mediated human gene therapy trials so far, have clearly pointed to the need for substantial improvement in the efficiency of AAV mediated transgene expression as well as the need to attenuate the capsid-or transgene specific innate or adaptive immune responses against these vectors to achieve successful long-term gene transfer. My research is thus focused on dissecting out the biology of AAV life-cycle by understanding the interactions between AAV and various host cellular proteins, use this knowledge to design strategies to either augment the efficiency of gene transfer or intervene with (immune response) processes which are detrimental to AAV’s survival, yet maintain the safety of these interventionist strategies to the host cellular environment and finally authenticate their use in therapeutic models such as pre-clinical animal models of haemophilia. My individual projects, were conceived to achieve this, albeit in parts, for an efficacious and safe gene therapy approach for haemophilia.

Ongoing Research Projects

• Swarnajayanti Fellow (PI: Jayandharan) Dec 2011- Dec 2016
  Department of Science and Technology, India 
  Optimized AAV serotype 2 and 5 vectors by bioengineering surface exposedmotifs to improve the efficacy of therapeutic gene transfer in hemophilia B.
• Early Career Investigator (PI: Jayandharan)  June 2010-June 2012  Bayer Inc, USA
  AAV vectors for the potential gene therapy of hemophilia B:  Modulation of the host immune response via the NF-kB pathway.
• Innovative  Young Biotechnologist Award  (PI: Jayandharan)  April 2011- March 2013  Department of Biotechnology (DBT), India
  Modulation of  Adeno-associated virus (AAV) replication by host cell transcriptional repressors: Pharmacologic and RNA interference to improve AAV vector delivery during gene therapy.
• Research grant (PI: Jayandharan, Co-I: Alok Srivastava, Sukesh C Nair) June 2011 June 2014 DBT, India
  Efficacy of bio-engineered adeno-associated virus serotype 8 vectors for the potential gene therapy of hemophilia A.