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Page 1
Selection of an Efficient AAV Vector for Robust CNS Transgene Expression.
Hanlon KS, Meltzer JC, Buzhdygan T, Cheng MJ, Sena-Esteves M, Bennett RE, Sullivan TP, Razmpour R, Gong Y, Ng C, Nammour J, Maiz D, Dujardin S, Ramirez SH, Hudry E, Maguire CA. Hanlon KS, et al. Among authors: ng c. Mol Ther Methods Clin Dev. 2019 Oct 23;15:320-332. doi: 10.1016/j.omtm.2019.10.007. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31788496 Free PMC article.
The AAV9 Variant Capsid AAV-F Mediates Widespread Transgene Expression in Nonhuman Primate Spinal Cord After Intrathecal Administration.
Beharry A, Gong Y, Kim JC, Hanlon KS, Nammour J, Hieber K, Eichler F, Cheng M, Stemmer-Rachamimov A, Stankovic KM, Welling DB, Ng C, Maguire CA. Beharry A, et al. Among authors: ng c. Hum Gene Ther. 2022 Jan;33(1-2):61-75. doi: 10.1089/hum.2021.069. Epub 2021 Aug 26. Hum Gene Ther. 2022. PMID: 34128391 Free PMC article.
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature.
Ramirez SH, Hale JF, McCarthy S, Cardenas CL, Dona KNUG, Hanlon KS, Hudry E, Cruz D, Ng C, Das S, Nguyen DM, Nammour J, Bennett RE, Andrews AM, Musolino PL, Maguire CA. Ramirez SH, et al. Among authors: ng c. Hum Gene Ther. 2023 Aug;34(15-16):682-696. doi: 10.1089/hum.2022.211. Hum Gene Ther. 2023. PMID: 37376759 Free PMC article.
In vivo selection in non-human primates identifies superior AAV capsids for on-target CSF delivery to spinal cord.
Hanlon KS, Cheng M, De La Cruz D, Patel N, Santoscoy MC, Gong Y, Ng C, Nguyen DM, Nammour J, Clark SW, Kozarsky K, Maguire CA. Hanlon KS, et al. Among authors: ng c. bioRxiv [Preprint]. 2023 Sep 13:2023.09.13.557506. doi: 10.1101/2023.09.13.557506. bioRxiv. 2023. Update in: Mol Ther. 2024 Aug 7;32(8):2584-2603. doi: 10.1016/j.ymthe.2024.05.040 PMID: 37745398 Free PMC article. Updated. Preprint.
In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord.
Hanlon KS, Cheng M, Ferrer RM, Ryu JR, Lee B, De La Cruz D, Patel N, Espinoza P, Santoscoy MC, Gong Y, Ng C, Nguyen DM, Nammour J, Clark SW, Heine VM, Sun W, Kozarsky K, Maguire CA. Hanlon KS, et al. Among authors: ng c. Mol Ther. 2024 Aug 7;32(8):2584-2603. doi: 10.1016/j.ymthe.2024.05.040. Epub 2024 Jun 5. Mol Ther. 2024. PMID: 38845196 Free PMC article.
An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs.
Vasireddy V, Maguire CA, Anderson DW, Ng C, Gong Y, Eichler F, Fourcade S, Guilera C, Onieva A, Sanchez A, Leal-Julià M, Verdés S, Dijkstra IME, Kemp S, Park H, Lutz T, Clark SW, Bosch A, Pujol A, Kozarsky K. Vasireddy V, et al. Among authors: ng c. Mol Ther Methods Clin Dev. 2024 Oct 9;32(4):101354. doi: 10.1016/j.omtm.2024.101354. eCollection 2024 Dec 12. Mol Ther Methods Clin Dev. 2024. PMID: 39524975 Free PMC article.
6,135 results