A GLB1 transgene with enhanced therapeutic potential for the preclinical development of ex-vivo gene therapy to treat mucopolysaccharidosis type IVB.
Crippa S, Alberti G, Passerini L, Savoia EO, Mancino M, De Ponti G, Santi L, Berti M, Testa M, Hernandez RJ, Quaranta P, Ceriotti S, Visigalli I, Morrone A, Paoli A, Forni C, Scala S, Degano M, Staiano L, Gregori S, Aiuti A, Bernardo ME.
Crippa S, et al. Among authors: de ponti g.
Mol Ther Methods Clin Dev. 2024 Aug 6;32(3):101313. doi: 10.1016/j.omtm.2024.101313. eCollection 2024 Sep 12.
Mol Ther Methods Clin Dev. 2024.
PMID: 39282079
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