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Page 1
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: nammour j. 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.
AAV-S: A versatile capsid variant for transduction of mouse and primate inner ear.
Ivanchenko MV, Hanlon KS, Hathaway DM, Klein AJ, Peters CW, Li Y, Tamvakologos PI, Nammour J, Maguire CA, Corey DP. Ivanchenko MV, et al. Among authors: nammour j. Mol Ther Methods Clin Dev. 2021 Mar 29;21:382-398. doi: 10.1016/j.omtm.2021.03.019. eCollection 2021 Jun 11. Mol Ther Methods Clin Dev. 2021. PMID: 33869656 Free PMC article.
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: nammour j. 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.
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: nammour j. 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.
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: nammour j. 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.
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: nammour j. 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.
Human CARMIL2 deficiency underlies a broader immunological and clinical phenotype than CD28 deficiency.
Lévy R, Gothe F, Momenilandi M, Magg T, Materna M, Peters P, Raedler J, Philippot Q, Rack-Hoch AL, Langlais D, Bourgey M, Lanz AL, Ogishi M, Rosain J, Martin E, Latour S, Vladikine N, Distefano M, Khan T, Rapaport F, Schulz MS, Holzer U, Fasth A, Sogkas G, Speckmann C, Troilo A, Bigley V, Roppelt A, Dinur-Schejter Y, Toker O, Bronken Martinsen KH, Sherkat R, Somekh I, Somech R, Shouval DS, Kühl JS, Ip W, McDermott EM, Cliffe L, Ozen A, Baris S, Rangarajan HG, Jouanguy E, Puel A, Bustamante J, Alyanakian MA, Fusaro M, Wang Y, Kong XF, Cobat A, Boutboul D, Castelle M, Aguilar C, Hermine O, Cheminant M, Suarez F, Yildiran A, Bousfiha A, Al-Mousa H, Alsohime F, Cagdas D, Abraham RS, Knutsen AP, Fevang B, Bhattad S, Kiykim A, Erman B, Arikoglu T, Unal E, Kumar A, Geier CB, Baumann U, Neven B; CARMIL2 Consortium; Rohlfs M, Walz C, Abel L, Malissen B, Marr N, Klein C, Casanova JL, Hauck F, Béziat V. Lévy R, et al. J Exp Med. 2023 Feb 6;220(2):e20220275. doi: 10.1084/jem.20220275. Epub 2022 Dec 14. J Exp Med. 2023. PMID: 36515678 Free PMC article.
Humans with inherited T cell CD28 deficiency are susceptible to skin papillomaviruses but are otherwise healthy.
Béziat V, Rapaport F, Hu J, Titeux M, Bonnet des Claustres M, Bourgey M, Griffin H, Bandet É, Ma CS, Sherkat R, Rokni-Zadeh H, Louis DM, Changi-Ashtiani M, Delmonte OM, Fukushima T, Habib T, Guennoun A, Khan T, Bender N, Rahman M, About F, Yang R, Rao G, Rouzaud C, Li J, Shearer D, Balogh K, Al Ali F, Ata M, Dabiri S, Momenilandi M, Nammour J, Alyanakian MA, Leruez-Ville M, Guenat D, Materna M, Marcot L, Vladikine N, Soret C, Vahidnezhad H, Youssefian L, Saeidian AH, Uitto J, Catherinot É, Navabi SS, Zarhrate M, Woodley DT, Jeljeli M, Abraham T, Belkaya S, Lorenzo L, Rosain J, Bayat M, Lanternier F, Lortholary O, Zakavi F, Gros P, Orth G, Abel L, Prétet JL, Fraitag S, Jouanguy E, Davis MM, Tangye SG, Notarangelo LD, Marr N, Waterboer T, Langlais D, Doorbar J, Hovnanian A, Christensen N, Bossuyt X, Shahrooei M, Casanova JL. Béziat V, et al. Among authors: nammour j. Cell. 2021 Jul 8;184(14):3812-3828.e30. doi: 10.1016/j.cell.2021.06.004. Epub 2021 Jul 1. Cell. 2021. PMID: 34214472 Free PMC article.
Correction: Dominant-negative mutations in human IL6ST underlie hyper-IgE syndrome.
Béziat V, Tavernier SJ, Chen YH, Ma CS, Materna M, Laurence A, Staal J, Aschenbrenner D, Roels L, Worley L, Claes K, Gartner L, Kohn LA, De Bruyne M, Schmitz-Abe K, Charbonnier LM, Keles S, Nammour J, Vladikine N, Luxman Maglorius Renkilaraj MR, Seeleuthner Y, Migaud M, Rosain J, Jeljeli M, Boisson B, Van Braeckel E, Rosenfeld JA, Dai H, Burrage LC, Murdock DR, Lambrecht BN, Avettand-Fenoel V, Vogel TP, Network UD, Esther CR, Haskologlu S, Dogu F, Ciznar P, Boutboul D, Ouachée-Chardin M, Amourette J, Lebras MN, Gauvain C, Tcherakian C, Ikinciogullari A, Beyaert R, Abel L, Milner JD, Grimbacher B, Couderc LJ, Butte MJ, Freeman AF, Catherinot É, Fieschi C, Chatila TA, Tangye SG, Uhlig HH, Haerynck F, Casanova JL, Puel A. Béziat V, et al. Among authors: nammour j. J Exp Med. 2020 Jun 1;217(7):e2019180405272020c. doi: 10.1084/jem.2019180405272020c. J Exp Med. 2020. PMID: 32516385 Free PMC article. No abstract available.
12 results