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Targeted Inactivation of the CCR5 HIV Coreceptor with Peptide Nucleic Acids

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R43AI104355-01
Agency Tracking Number: R43AI104355
Amount: $250,298.00
Phase: Phase I
Program: SBIR
Solicitation Topic Code: NIAID
Solicitation Number: PA12-088
Solicitation Year: 2014
Award Year: 2014
Award Start Date (Proposal Award Date): N/A
Award End Date (Contract End Date): N/A
Small Business Information
BOX 8175
NEW HAVEN, CT 06530-0175
United States
DUNS: 142406110
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (203) 503-0383
Business Contact
Phone: (203) 494-5288
Research Institution

DESCRIPTION (provided by applicant): Over 34 million people are currently living with HIV/AIDS. Combination antiviral therapies have significantly reduced the morbidity and mortality of the disease; however, there is currently no cure for the disease. Discovery that a naturally occurring variant of CCR5, the delta32 mutation, renders cells resistant to HIV infection has led to efforts by several academic laboratories and biotech companies to find gene therapy approaches to mimic the delta32 mutation. This project will demonstrate the feasibility of using peptide nucleic acids (PNAs) as a therapeutic to genetically truncate the CCR5 protein in CD34+ stem cells and block HIV cell entry. Preliminary experiments that modified the CCR5 gene used a targeting PNA with a donor DNA sequence to induce a stop codon near the delta32 mutation. In this Phase I project, we will develop a novel strategy for PNA-induced CCR5 gene modification to increase the CD34+ mutation frequency to clinically useful levels. Specific

* Information listed above is at the time of submission. *

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