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Active immunotherapy and alternative therapeutic modalities for Alzheimer's disease.
Weninger S, Sperling B, Alexander R, Ivarsson M, Menzies FM, Powchik P, Weber CJ, Altar CA, Crystal RG, Haggarty SJ, Loring J, Bain LJ, Carrillo MC. Weninger S, et al. Among authors: crystal rg. Alzheimers Dement (N Y). 2020 Oct 12;6(1):e12090. doi: 10.1002/trc2.12090. eCollection 2020. Alzheimers Dement (N Y). 2020. PMID: 33083513 Free PMC article.
Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway.
Lojewski X, Staropoli JF, Biswas-Legrand S, Simas AM, Haliw L, Selig MK, Coppel SH, Goss KA, Petcherski A, Chandrachud U, Sheridan SD, Lucente D, Sims KB, Gusella JF, Sondhi D, Crystal RG, Reinhardt P, Sterneckert J, Schöler H, Haggarty SJ, Storch A, Hermann A, Cotman SL. Lojewski X, et al. Among authors: crystal rg. Hum Mol Genet. 2014 Apr 15;23(8):2005-22. doi: 10.1093/hmg/ddt596. Epub 2013 Nov 23. Hum Mol Genet. 2014. PMID: 24271013 Free PMC article.
Advances in the Treatment of Neuronal Ceroid Lipofuscinosis.
Rosenberg JB, Chen A, Kaminsky SM, Crystal RG, Sondhi D. Rosenberg JB, et al. Among authors: crystal rg. 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.
Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis.
Crystal RG, Sondhi D, Hackett NR, Kaminsky SM, Worgall S, Stieg P, Souweidane M, Hosain S, Heier L, Ballon D, Dinner M, Wisniewski K, Kaplitt M, Greenwald BM, Howell JD, Strybing K, Dyke J, Voss H. Crystal RG, et al. Hum Gene Ther. 2004 Nov;15(11):1131-54. doi: 10.1089/hum.2004.15.1131. Hum Gene Ther. 2004. PMID: 15610613 Clinical Trial.
895 results