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Page 1
Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders.
Sci Adv. 2022 Sep 23;8(38):eabn4704. doi: 10.1126/sciadv.abn4704. Epub 2022 Sep 21.
Sci Adv. 2022.
PMID: 36129972
Free PMC article.
Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy.
Muñoz S, Bertolin J, Jimenez V, Jaén ML, Garcia M, Pujol A, Vilà L, Sacristan V, Barbon E, Ronzitti G, El Andari J, Tulalamba W, Pham QH, Ruberte J, VandenDriessche T, Chuah MK, Grimm D, Mingozzi F, Bosch F.
Muñoz S, et al. Among authors: pham qh.
Mol Metab. 2024 Mar;81:101899. doi: 10.1016/j.molmet.2024.101899. Epub 2024 Feb 10.
Mol Metab. 2024.
PMID: 38346589
Free PMC article.
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Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors.
Tulalamba W, Weinmann J, Pham QH, El Andari J, VandenDriessche T, Chuah MK, Grimm D.
Tulalamba W, et al. Among authors: pham qh.
Gene Ther. 2020 Apr;27(3-4):170-179. doi: 10.1038/s41434-019-0106-3. Epub 2019 Oct 17.
Gene Ther. 2020.
PMID: 31624368
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Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua.
Nair N, De Wolf D, Nguyen PA, Pham QH, Samara-Kuko E, Landau J, Blouse GE, Chuah MK, VandenDriessche T.
Nair N, et al. Among authors: pham qh.
Blood. 2021 May 27;137(21):2902-2906. doi: 10.1182/blood.2020006005.
Blood. 2021.
PMID: 33735915
Free PMC article.
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