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Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse m…
Balakrishnan B; Yan X; McCue MD; Bellagamba O; Guo A; Winkler F; Thall J; Crawford L; Dimen R; Chen S; McEnaney S; Wu Y; Zimmer M; Sarkis J; Martini PGV; Finn PF; Lai K.
Mol Ther Methods Clin Dev
(Unknown)
Published: 2024
AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich's ataxia.
Chang JC; Ryan MR; Stark MC; Liu S; Purushothaman P; Bolan F; Johnson CA; Champe M; Meng H; Lawlor MW; Halawani S; Ngaba LV; Lynch DR; Davis C; Gonzalo-Gil E; Lutz C; Urbinati F; Medicherla B; Fonck C.
Mol Ther Methods Clin Dev
(Unknown)
Published: 2024
Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency.
Mével M; Pichard V; Bouzelha M; Alvarez-Dorta D; Lalys PA; Provost N; Allais M; Mendes A; Landagaray E; Ducloyer JB; Toublanc E; Galy A; Brument N; Lefevre GM; Gouin SG; Isiegas C; Le Meur G; Cronin T; Le Guiner C; Weber M; Moullier P; Ayuso E; Deniaud D; Adjali O.
Mol Ther Methods Clin Dev
(Unknown)
Published: 2024
Long-term stability of clinical-grade lentiviral vectors for cell therapy.
Jadlowsky JK; Leskowitz R; McKenna S; Karar J; Ma Y; Dai A; Plesa G; Chen F; Alexander K; Petrella J; Gong N; Hwang WT; Farrelly O; Barber-Rotenberg J; Christensen S; Gonzalez VE; Chew A; Fraietta JA; June CH.
Mol Ther Methods Clin Dev
(Unknown)
Published: 2024
Manufacture of CD22 CAR T cells following positive versus negative selection results in distinct cytokine secretion pro…
Song HW; Benzaoui M; Dwivedi A; Underwood S; Shao L; Achar S; Posarac V; Remley VA; Prochazkova M; Cai Y; Jin P; Somerville RP; Stroncek DF; Altan-Bonnet G; Shah NN; Chien CD; Taylor N; Highfill SL.
Mol Ther Methods Clin Dev
(Unknown)
Published: 2023
Regnase-1 overexpression as a therapeutic approach of Marfan syndrome.
Noormalal M; Schmiedel N; Bozoglu T; Matzen A; Hille S; Basha DI; Vijaya Shetty PM; Wolf A; Zaradzki M; Arif R; Pühler T; Lutter G; Wagner AH; Kupatt C; Frank D; Frey N; Remes A; Müller OJ.
Mol Ther Methods Clin Dev
(Unknown)
Published: 2023
Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog.
Van Gorder L; Doshi BS; Willis E; Nichols TC; Cook E; Everett JK; Merricks EP; Arruda VR; Bushman FD; Callan MB; Samelson-Jones BJ.
Mol Ther Methods Clin Dev
(Unknown)
Published: 2023
CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crig…
Bortolussi G; Iaconcig A; Canarutto G; Porro F; Ferrucci F; Galletta C; Díaz-Muñoz C; Rawat V; De Caneva A; Olajide OJ; Zentilin L; Piazza S; Bočkor L; Muro AF.
Mol Ther Methods Clin Dev
(Unknown)
Published: 2023
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy fo…
Catalano F; Vlaar EC; Katsavelis D; Dammou Z; Huizer TF; van den Bosch JC; Hoogeveen-Westerveld M; van den Hout HJMP; Oussoren E; Ruijter GJG; Schaaf G; Pike-Overzet K; Staal FJT; van der Ploeg AT; Pijnappel WWMP.
Mol Ther Methods Clin Dev
(Unknown)
Published: 2023