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Benjamin P Kleinstiver

Explore the profile of Benjamin P Kleinstiver including associated specialties, affiliations and a list of published articles. Areas
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Articles 78
Citations 5924
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Recent Articles
11.
Fry M, Navarro P, Hakim P, Ananda V, Qin X, Landoni J, et al.
EMBO J . 2024 Jan; 43(3):391-413. PMID: 38225406
Cristae membrane state plays a central role in regulating mitochondrial function and cellular metabolism. The protein Optic atrophy 1 (Opa1) is an important crista remodeler that exists as two forms...
12.
Alves C, Ha L, Yaworski R, Sutton E, Lazzarotto C, Christie K, et al.
Nat Biomed Eng . 2023 Dec; 8(2):118-131. PMID: 38057426
Spinal muscular atrophy (SMA) is caused by mutations in SMN1. SMN2 is a paralogous gene with a C•G-to-T•A transition in exon 7, which causes this exon to be skipped in...
13.
Zhao L, Koseki S, Silverstein R, Amrani N, Peng C, Kramme C, et al.
Nat Commun . 2023 Oct; 14(1):6175. PMID: 37794046
CRISPR enzymes require a defined protospacer adjacent motif (PAM) flanking a guide RNA-programmed target site, limiting their sequence accessibility for robust genome editing applications. In this study, we recombine the...
14.
Ferreira da Silva J, Tou C, King E, Eller M, Ma L, Rufino-Ramos D, et al.
bioRxiv . 2023 Sep; PMID: 37745481
Genome editing technologies that install diverse edits can widely enable genetic studies and new therapeutics. Here we develop click editing, a genome writing platform that couples the advantageous properties of...
15.
Tao Y, Lamas V, Du W, Zhu W, Li Y, Whittaker M, et al.
Nat Commun . 2023 Aug; 14(1):4928. PMID: 37582836
Mutations in Atp2b2, an outer hair cell gene, cause dominant hearing loss in humans. Using a mouse model Atp2b2, with a dominant hearing loss mutation (Oblivion), we show that liposome-mediated...
16.
McLean Z, Gao D, Correia K, Roy J, Shibata S, Farnum I, et al.
bioRxiv . 2023 Aug; PMID: 37547003
Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder whose motor, cognitive, and behavioral manifestations are caused by an expanded, somatically unstable CAG repeat in the first exon of that...
17.
Mesaki K, Juvet S, Yeung J, Guan Z, Wilson G, Hu J, et al.
J Heart Lung Transplant . 2023 Jun; 42(10):1363-1377. PMID: 37315746
Background: Inflammatory injury in the donor lung remains a persistent challenge in lung transplantation that limits donor organ usage and post-transplant outcomes. Inducing immunomodulatory capacity in donor organs could address...
18.
Peters C, Hanlon K, Ivanchenko M, Zinn E, Linarte E, Li Y, et al.
Mol Ther . 2023 Jun; 31(8):2439-2453. PMID: 37312453
Usher syndrome type 1F (USH1F), characterized by congenital lack of hearing and balance and progressive loss of vision, is caused by mutations in the PCDH15 gene. In the Ashkenazi population,...
19.
Choi D, Shin J, Zeng S, Hong E, Jang J, Loupe J, et al.
bioRxiv . 2023 May; PMID: 37162872
An expanded CAG repeat in the huntingtin gene ( ) causes Huntington's disease (HD). Since the length of uninterrupted CAG repeat, not polyglutamine, determines the age-at-onset in HD, base editing...
20.
Kiss M, Mindur J, Yates A, Lee D, Fullard J, Anzai A, et al.
Immunity . 2023 May; 56(7):1502-1514.e8. PMID: 37160117
Glial cells and central nervous system (CNS)-infiltrating leukocytes contribute to multiple sclerosis (MS). However, the networks that govern crosstalk among these ontologically distinct populations remain unclear. Here, we show that,...