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Persistent gene therapy in muscle may not require immunosuppression

Date:
November 15, 2013
Source:
Journal of Clinical Investigation
Summary:
In a new study, researchers evaluated the persistence of rAAV-mediated expression the gene encoding M-type -1 antitrypsin in patients that were AAT deficient

Successful gene therapy is based on the effective delivery and maintained expression of healthy copies of a gene into tissues of individuals with a disease-associated genetic mutation. Recombinant adeno-associated virus (rAAV) vectors have shown promise in early clinical trials as effective therapies for several genetic diseases, including Leber congenital amaurosis, Parkinson disease, and hemophilia. Unfortunately, delivery of rAAV vectors to tissues other than the retina and CNS often results in development of an immune response against the viral capsid. The development of a neutralizing response against the rAAV vector prevents sustained expression of the healthy gene in the absence of immunosuppression.

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In this issue of the Journal of Clinical Investigation, Christian Mueller and colleagues at the University of Massachusetts Medical School evaluated the persistence of rAAV-mediated expression the gene encoding M-type α-1 antitrypsin (M-AAT) in patients that were AAT deficient. Patients received multiple intramuscular doses without immunosuppression, and M-ATT expression was evaluated in muscle biopsies. The authors determined that subjects sustained M-ATT expression in muscle tissue for at least one year, despite an initial influx of immune cells. Further evaluation of muscle fibers revealed a substantial population of regulatory T cells in patients with persistent M-ATT expression.

Together, the results from this study suggests that delivery of an M-ATT-encoding rAAV vector promotes a regulatory immune response that allows for long term gene expression that does not require immune suppression.


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The above story is based on materials provided by Journal of Clinical Investigation. Note: Materials may be edited for content and length.


Journal Reference:

  1. Christian Mueller, Jeffrey D. Chulay, Bruce C. Trapnell, Margaret Humphries, Brenna Carey, Robert A. Sandhaus, Noel G. McElvaney, Louis Messina, Qiushi Tang, Farshid N. Rouhani, Martha Campbell-Thompson, Ann Dongtao Fu, Anthony Yachnis, David R. Knop, Guo-jie Ye, Mark Brantly, Roberto Calcedo, Suryanarayan Somanathan, Lee P. Richman, Robert H. Vonderheide, Maigan A. Hulme, Todd M. Brusko, James M. Wilson, Terence R. Flotte. Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression. Journal of Clinical Investigation, 2013; DOI: 10.1172/JCI70314

Cite This Page:

Journal of Clinical Investigation. "Persistent gene therapy in muscle may not require immunosuppression." ScienceDaily. ScienceDaily, 15 November 2013. <www.sciencedaily.com/releases/2013/11/131115130201.htm>.
Journal of Clinical Investigation. (2013, November 15). Persistent gene therapy in muscle may not require immunosuppression. ScienceDaily. Retrieved January 24, 2015 from www.sciencedaily.com/releases/2013/11/131115130201.htm
Journal of Clinical Investigation. "Persistent gene therapy in muscle may not require immunosuppression." ScienceDaily. www.sciencedaily.com/releases/2013/11/131115130201.htm (accessed January 24, 2015).

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