Therapeutic Candidate or Device AAV9 Gene Therapy For An Ultra-Rare Disease Called CMT4J Indication CMT4J or the FIG4 Gene Therapeutic Mechanism Gene Therapy through an Intrathecal One-Time Injection Unmet Medical…
Therapeutic Candidate or Device Hematopoietic stem/progenitor cells collected from patients with hypophosphatasia and genetically modified with a lentiviral vector to release TNALP Indication Hypophosphatasia (HPP) Therapeutic Mechanism We are proposing…
Therapeutic Candidate or Device Autologous blood stem cells edited to restore iduronidase expression Indication Severe Mucopolysaccharidosis Type 1 (MPS1/ Hurler's syndrome) Therapeutic Mechanism Autologous blood stem cells undergo genome editing…
Therapeutic Candidate or Device Transduced Hematopoietic Stem Cells from Peripheral Blood Stem Cells of adults and pediatric patients with cystinosis Indication Autologous hematopoietic stem cell gene therapy for patients with…
*Please note: this video contains graphic images of a disfiguring disease John Wagner presented the closing scientific talk at the 2011 CIRM Grantee Meeting. Weaving together stories of medical history,…
Spotlight on Disease Team Awards - Genetic Skin Disease: Welcoming Remarks Robert Klein J.D., gave the welcoming remarks for the "Spotlight on Disease Team Awards: Genetic Skin Disease," an educational…
Spotlight on Disease Team Awards - Genetic Skin Disease: Introduction Phillip Pizzo, M.D., spoke at the "Spotlight on Disease Team Awards: Genetic Skin Disease," an educational event presented at the…
Spotlight on Disease Team Awards - Genetic Skin Disease: Lynn Anderson Lynn Anderson, spoke at the "Spotlight on Disease Team Awards: Genetic Skin Disease," an educational event presented at the…
Spotlight on Disease Team Awards - Genetic Skin Disease: Albert Lane Albert Lane, M.D., spoke at the "Spotlight on Disease Team Awards: Genetic Skin Disease," an educational event presented at…
Spotlight on Disease Team Awards - Genetic Skin Disease: Marius Wernig Marius Wernig, M.D., spoke at the "Spotlight on Disease Team Awards: Genetic Skin Disease," an educational event presented at…
