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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: crystal rg. 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.
Population studies have shown that an inheritance of the apolipoprotein E (APOE) variant APOE4 allele increases the risk of developing AD, whereas APOE2 homozygotes are protected from late-onset AD. ...
Population studies have shown that an inheritance of the apolipoprotein E (APOE) variant APOE4 allele increases the risk of developin …
Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-Modified AAV Vector.
Karan KR, Andrzejewski S, Stiles KM, Hackett NR, Crystal RG. Karan KR, et al. Among authors: crystal rg. Hum Gene Ther. 2024 Nov;35(21-22):904-916. doi: 10.1089/hum.2024.112. Epub 2024 Oct 16. Hum Gene Ther. 2024. PMID: 39318239
We hypothesized that APOE4 could be suppressed in the CNS of APOE4 homozygotes using adeno-associated virus (AAV) expression of microRNAs (miRNA) designed to hybridize to APOE mRNA. We screened nine different miRNAs targeting APOE following transfection in HEK293T a …
We hypothesized that APOE4 could be suppressed in the CNS of APOE4 homozygotes using adeno-associated virus (AAV) expression of microRNAs (m …
Prevalence of the apolipoprotein E Arg145Cys dyslipidemia at-risk polymorphism in African-derived populations.
Abou Ziki MD, Strulovici-Barel Y, Hackett NR, Rodriguez-Flores JL, Mezey JG, Salit J, Radisch S, Hollmann C, Chouchane L, Malek J, Zirie MA, Jayyuosi A, Gotto AM Jr, Crystal RG. Abou Ziki MD, et al. Among authors: crystal rg. Am J Cardiol. 2014 Jan 15;113(2):302-8. doi: 10.1016/j.amjcard.2013.09.021. Epub 2013 Oct 3. Am J Cardiol. 2014. PMID: 24239320 Free PMC article.
Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models.
Zhao L, Gottesdiener AJ, Parmar M, Li M, Kaminsky SM, Chiuchiolo MJ, Sondhi D, Sullivan PM, Holtzman DM, Crystal RG, Paul SM. Zhao L, et al. Among authors: crystal rg. Neurobiol Aging. 2016 Aug;44:159-172. doi: 10.1016/j.neurobiolaging.2016.04.020. Epub 2016 Apr 30. Neurobiol Aging. 2016. PMID: 27318144
Preclinical and clinical studies have shown that apolipoprotein E (APOE) genotype also predicts the timing and amount of brain amyloid-beta (Abeta) peptide deposition and amyloid burden (epsilon4 >epsilon3 >epsilon2). Using several administration protocols, we now re …
Preclinical and clinical studies have shown that apolipoprotein E (APOE) genotype also predicts the timing and amount of brain amyloi …
Prime Editing of Alzheimer's Disease High-Risk APOE4 Allele by Brain-Directed Adeno-Associated Virus Vectors.
Günaydin C, Hackett NR, Wakim V, Sondhi D, Kaminsky SM, Crystal RG. Günaydin C, et al. Among authors: crystal rg. Hum Gene Ther. 2026 Feb 8:10430342251401888. doi: 10.1177/10430342251401888. Online ahead of print. Hum Gene Ther. 2026. PMID: 41449667
Common variants of the apolipoprotein E (APOE) gene have a major impact on the risk of developing Alzheimer's disease (AD). Relative to homozygotes with the common E3 allele, the APOE4 variant (C112R) increases risk by 3.5-fold in E3/E4 heterozygotes and 15-fold in E4 homo …
Common variants of the apolipoprotein E (APOE) gene have a major impact on the risk of developing Alzheimer's disease (AD). Relative …