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Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing.
Nat Commun. 2022 Oct 10;13(1):5947. doi: 10.1038/s41467-022-33745-4.
Nat Commun. 2022.
PMID: 36210364
Free PMC article.
What's new and what's next for gene therapy in Pompe disease?
Roger AL, Sethi R, Huston ML, Scarrow E, Bao-Dai J, Lai E, Biswas DD, El Haddad L, Strickland LM, Kishnani PS, ElMallah MK.
Roger AL, et al.
Expert Opin Biol Ther. 2022 Sep;22(9):1117-1135. doi: 10.1080/14712598.2022.2067476. Epub 2022 Apr 27.
Expert Opin Biol Ther. 2022.
PMID: 35428407
Free PMC article.
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Breathing in Duchenne muscular dystrophy: translation to therapy.
Mhandire DZ, Burns DP, Roger AL, O'Halloran KD, ElMallah MK.
Mhandire DZ, et al. Among authors: roger al.
J Physiol. 2022 Aug;600(15):3465-3482. doi: 10.1113/JP281671. Epub 2022 Jun 24.
J Physiol. 2022.
PMID: 35620971
Free PMC article.
Review.
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GAA deficiency disrupts distal airway cells in Pompe disease.
El Haddad L, Lai E, Murthy PKL, Biswas DD, Soufny R, Roger AL, Tata PR, ElMallah MK.
El Haddad L, et al. Among authors: roger al.
Am J Physiol Lung Cell Mol Physiol. 2023 Sep 1;325(3):L288-L298. doi: 10.1152/ajplung.00032.2023. Epub 2023 Jun 27.
Am J Physiol Lung Cell Mol Physiol. 2023.
PMID: 37366541
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