TELOMERASE REVERSE TRANSCRIPTASE-BASED THERAPIES
The invention provides compositions and methods useful for the treatment and prevention of conditions associated with short telomere length.
1 .- 12 . (canceled)
13 . A method of treating a condition associated with a disease, the method comprising:
administering a recombinant viral vector comprising at least a capsid and a nucleic acid comprising a coding sequence for telomerase reverse transcriptase (TERT) to a subject, wherein the condition is associated with pulmonary fibrosis.
14 . The method of claim 13 , wherein TERT is encoded by a nucleic acid sequence comprising a sequence that is at least 60% identical to the sequence of SEQ ID NO: 1 or SEQ ID NO: 3.
15 . The method of claim 13 , wherein TERT comprises an amino acid sequence that is at least 60% identical to the amino acid sequence of SEQ ID NO:2 or SEQ ID NO: 4.
16 . The method of claim 13 , wherein the nucleic acid sequence encoding TERT is operably linked to a regulatory sequence that drives the expression of the coding sequence.
17 . The method of claim 13 , wherein the vector is a non-integrative vector.
18 . The method of claim 13 , wherein the vector is an adeno-associated virus-based non-integrative vector.
19 . The method of claim 13 , wherein the vector is an adeno-associated virus-based vector whose capsid is derived from a serotype 9 adeno-associated virus (AAV9).
20 . The method of claim 19 , wherein the nucleic acid sequence packaged in the capsid is flanked at both ends by internal terminal repeats of the serotype 2 adeno-associated virus.
21 . The method of claim 13 , wherein the vector comprises a regulatory sequence which is a constitutive promoter.
22 . The method of claim 21 , wherein the constitutive promoter is the cytomegalovirus (CMV) promoter.
23 . The method of claim 13 , wherein the pulmonary fibrosis is characterized by mutations in a gene or genes involved in telomere maintenance.