Publication
| Comper F, Miranda CJ, Liou B, Dodev T, Jeyakumar JM, Canavese M, Cocita C, Khoshrou K, Tiscornia G, Chisari E, Stotter E, Shehu E, Sridharan S, Yu IM, Pandya J, Khinder J, Northcott N, Kalcheva P, Correia S, Sun Y, Dane AP, Sheridan R, Nathwani AC, Corbau R, FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1 Molecular therapy : the journal of the American Society of Gene Therapy33:3789-3807 2025 |
| PubMed ID: 40340248 |
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| Records Return:
(2)
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| Catalog ID | Sex | Age at Sampling | Description | Collection_Type_id |
| GM01260 | Female | 11 MO | GAUCHER DISEASE, TYPE II | GM |
| GM00372 | Male | 29 YR | GAUCHER DISEASE, TYPE I | GM |
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