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Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2.
Sondhi D, Kaminsky SM, Hackett NR, Pagovich OE, Rosenberg JB, De BP, Chen A, Van de Graaf B, Mezey JG, Mammen GW, Mancenido D, Xu F, Kosofsky B, Yohay K, Worgall S, Kaner RJ, Souwedaine M, Greenwald BM, Kaplitt M, Dyke JP, Ballon DJ, Heier LA, Kiss S, Crystal RG. Sondhi D, et al. Among authors: chen a. Sci Transl Med. 2020 Dec 2;12(572):eabb5413. doi: 10.1126/scitranslmed.abb5413. Sci Transl Med. 2020. PMID: 33268510 Free PMC article. Clinical Trial.
Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene.
Sondhi D, Scott EC, Chen A, Hackett NR, Wong AM, Kubiak A, Nelvagal HR, Pearse Y, Cotman SL, Cooper JD, Crystal RG. Sondhi D, et al. Among authors: chen a. Hum Gene Ther. 2014 Mar;25(3):223-39. doi: 10.1089/hum.2012.253. Epub 2014 Mar 4. Hum Gene Ther. 2014. PMID: 24372003 Free PMC article.
Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates.
Rosenberg JB, Sondhi D, Rubin DG, Monette S, Chen A, Cram S, De BP, Kaminsky SM, Sevin C, Aubourg P, Crystal RG. Rosenberg JB, et al. Among authors: chen a. Hum Gene Ther Clin Dev. 2014 Sep;25(3):164-77. doi: 10.1089/humc.2013.239. Epub 2014 Aug 21. Hum Gene Ther Clin Dev. 2014. PMID: 25144894 Free PMC article.
AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease.
Rosenberg JB, Kaplitt MG, De BP, Chen A, Flagiello T, Salami C, Pey E, Zhao L, Ricart Arbona RJ, Monette S, Dyke JP, Ballon DJ, Kaminsky SM, Sondhi D, Petsko GA, Paul SM, Crystal RG. Rosenberg JB, et al. Among authors: chen a. Hum Gene Ther Clin Dev. 2018 Mar;29(1):24-47. doi: 10.1089/humc.2017.231. Epub 2018 Mar 13. Hum Gene Ther Clin Dev. 2018. PMID: 29409358 Free PMC article.
Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy.
Sacramento CB, Sondhi D, Rosenberg JB, Chen A, Giordano S, Pey E, Lee V, Stiles KM, Havlicek DF, Leopold PL, Kaminsky SM, Crystal RG. Sacramento CB, et al. Among authors: chen a. Hum Gene Ther. 2020 Jan;31(1-2):57-69. doi: 10.1089/hum.2019.174. Epub 2019 Dec 13. Hum Gene Ther. 2020. PMID: 31608704
Advances in the Treatment of Neuronal Ceroid Lipofuscinosis.
Rosenberg JB, Chen A, Kaminsky SM, Crystal RG, Sondhi D. Rosenberg JB, et al. Among authors: chen a. Expert Opin Orphan Drugs. 2019;7(11):473-500. doi: 10.1080/21678707.2019.1684258. Epub 2019 Nov 27. Expert Opin Orphan Drugs. 2019. PMID: 33365208 Free PMC article.
Safety of Intravenous Administration of an AAV8 Vector Coding for an Oxidation-Resistant Human α1-Antitrypsin for the Treatment of α1-Antitrypsin Deficiency.
Rosenberg JB, De BP, Greco A, Gorman N, Kooner V, Chen A, Yost-Bido M, Munoz-Zuluaga C, Kaminsky SM, Rostami M, Monette S, Crystal RG, Sondhi D. Rosenberg JB, et al. Among authors: chen a. Hum Gene Ther. 2023 Feb;34(3-4):139-149. doi: 10.1089/hum.2022.192. Hum Gene Ther. 2023. PMID: 36606685 Free PMC article.
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