Benjamin P Kleinstiver
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Explore the profile of Benjamin P Kleinstiver including associated specialties, affiliations and a list of published articles.
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78
Citations
5924
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Recent Articles
1.
Yun S, Chekuri A, Art J, Kondabolu K, Slaugenhaupt S, Zeltner N, et al.
bioRxiv
. 2024 Dec;
PMID: 39651221
Familial dysautonomia (FD) is a fatal autosomal recessive congenital neuropathy caused by a T-to-C mutation in intron 20 of the () gene, which causes tissue-specific skipping of exon 20 and...
2.
Alves C, Das S, Krishnan V, Ha L, Fox L, Stutzman H, et al.
bioRxiv
. 2024 Nov;
PMID: 39605323
Genetic vascular disorders are prevalent diseases that have diverse etiologies and few treatment options. Pathogenic missense mutations in the alpha actin isotype 2 gene () primarily affect smooth muscle cell...
3.
Nieland L, Vrijmoet A, Jetten I, Rufino-Ramos D, de Reus A, Breyne K, et al.
Mol Ther
. 2024 Nov;
33(1):133-151.
PMID: 39563028
Glioblastoma (GB), the most aggressive tumor of the central nervous system (CNS), has poor patient outcomes with limited effective treatments available. MicroRNA-21 (miR-21(a)) is a known oncogene, abundantly expressed in...
4.
Huynh P, Hoffmann J, Gerhardt T, Kiss M, Zuraikat F, Cohen O, et al.
Nature
. 2024 Oct;
635(8037):168-177.
PMID: 39478215
Sleep is integral to cardiovascular health. Yet, the circuits that connect cardiovascular pathology and sleep are incompletely understood. It remains unclear whether cardiac injury influences sleep and whether sleep-mediated neural...
5.
Bzhilyanskaya V, Ma L, Liu S, Fox L, Whittaker M, Meis R, et al.
Sci Transl Med
. 2024 Oct;
16(769):eadj6779.
PMID: 39413163
X-linked chronic granulomatous disease (X-CGD) is an inborn error of immunity (IEI) resulting from genetic mutations in the cytochrome b-245 beta chain () gene. The applicability of base editors (BEs)...
6.
Ferreira da Silva J, Tou C, King E, Eller M, Rufino-Ramos D, Ma L, et al.
Nat Biotechnol
. 2024 Jul;
PMID: 39039307
Genome editing technologies based on DNA-dependent polymerases (DDPs) could offer several benefits compared with other types of editors to install diverse edits. Here, we develop click editing, a genome writing...
7.
8.
Leandro K, Rufino-Ramos D, Breyne K, Di Ianni E, Lopes S, Nobre R, et al.
Adv Drug Deliv Rev
. 2024 Jun;
211:115346.
PMID: 38849005
Gene editing technologies have the potential to correct genetic disorders by modifying, inserting, or deleting specific DNA sequences or genes, paving the way for a new class of genetic therapies....
9.
Mesaki K, Yamamoto H, Juvet S, Yeung J, Guan Z, Akhter A, et al.
Hum Gene Ther
. 2024 May;
35(11-12):374-387.
PMID: 38717950
The ongoing advancements in CRISPR-Cas technologies can significantly accelerate the preclinical development of both and organ genome-editing therapeutics. One of the promising applications is to genetically modify donor organs prior...
10.
McLean Z, Gao D, Correia K, Roy J, Shibata S, Farnum I, et al.
Nat Commun
. 2024 Apr;
15(1):3182.
PMID: 38609352
Huntington's disease (HD) is a dominant neurological disorder caused by an expanded HTT exon 1 CAG repeat that lengthens huntingtin's polyglutamine tract. Lowering mutant huntingtin has been proposed for treating...