Award
National Heart, Lung, and Blood Institute 5R01HL172489-03
In vivo hematopoietic stem cell genetic modification by viral particles
Recipient
Boston Children's Hospital
Award Amount
$842,256.00
Ceiling
$842,256.00
Awarded
August 04, 2026
Identifier
5R01HL172489-03
The award supports development of an innovative in vivo gene therapy platform for hematopoietic stem cells to treat genetic blood disorders, aiming to simplify treatment and reduce risks compared to current ex vivo methods.
Description
This project aims to develop a novel gene therapy approach that can be administered directly to a patient’s blood stem cells via vein, avoiding stem cell collection, complex cell manufacture, and toxic chemotherapy. The project will develop integrating vectors for in vivo delivery to treat X-SCID, engineer virus-like particles for Cas9 delivery into quiescent HSCs to treat SCD, and modify particles for base editor transfer to correct mutations underlying Shwachman-Diamond Syndrome.