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US 11,529,396 B2Granted
Telomerase reverse transcriptase-based therapies
AssigneesFundacióN Del Sector PúBlico Estatal Centro Nacional Dl'investigaciones OncolóGicas Carlos III (F.S.P. Cnio), Universitat Autonoma De Barcelona
InventorsMaria Bobadilla, Ivan Formentini, Maria Antonia Blasco Marhuenda, Christian Baer, Fàtima Bosch I Tubert
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Abstract
The invention provides compositions and methods useful for the treatment and prevention of conditions associated with short telomere length.
Claims7
A method of treating a condition associated with aplastic anemia, the method comprising:
- administering to a subject in need thereof an adeno-associated virus-based vector comprising at least a capsid and a nucleic acid encoding telomerase reverse transcriptase (TERT) operably linked to a regulatory sequence that drives the expression of the encoded TERT, wherein the method results in transduction of hematopoietic stem cells.
Dependencies are inferred from the claim text.