NIH R01 · 2024
Cystic Fibrosis Gene Therapy with adeno-associated viral vectors
Abstract: We and others have shown that AAV vectors have great potential as gene therapeutic agents (3-5), in particular for CF. Studies originating from our group led to the first use of rAAV in humans (7). Many pre- clinical and clinical trials have shown that AAV vectors can be used safely (5,6,9). The major challenge, however, is that they have not achieved a reproducible therapeutic effect, making it necessary to take a new approach to AAV gene therapy. In previous work, we have identified three new strategies to alleviate these problems: 1) the use of AAV1, which is more tropic for the lung; 2) use of 27-264, a truncated version of CFTR that corrects F508 by a novel mechanism; and 3)…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.