Complete Knockdown Of Gene Inhibits HIV Infection

The CCR5 co-receptor is essential for the entry of HIV into cells but is dispensable for normal cellular functions and therefore an ideal therapeutic target - a subgroup of the human population that have a defective CCR5 gene are physiologically normal but are known to be resistant to HIV infection and disease progression. In the present study, Ramesh Akkina and Joseph Anderson derived several highly potent siRNAs capable of complete suppression of CCR5 expression. When these siRNAs were introduced into transgenic white blood cells produced from cultured stem cells and challenged with a predominantly transmitted R5-tropic strain of HIV-1, remarkable resistance to viral infection was observed. This suggests these anti-CCR5 siRNAs are among the most effective demonstrated to date and are very promising candidates for clinical applications.

According to the authors, "For innovative AIDS therapies to succeed in the long term, novel strategies need to be employed. In this regard, gene therapy approaches that focus on cellular molecules involved in viral attachment have great potential". They propose that the use of CCR5 siRNAs, in combination with siRNAs directed against T cell tropic viruses and delivered via a single gene transfer vector to modify blood cells, might confer broad anti-viral protection against HIV.

Author contact:

Ramesh Akkina (Colorado State University, Fort Collins, CO, USA)
E-mail: akkina@colostate.edu

(C) Gene Therapy press release.

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