Results 171 to 180 of about 179,984 (263)

Monoallelic POLR3A Variants Cause Early‐Onset Peripheral Neuropathy

open access: yesAnnals of Neurology, EarlyView.
Objective Biallelic variants in genes encoding the RNA polymerase III complex (Pol III) cause a spectrum of neurological disorders primarily affecting the central nervous system. Monoallelic variants have been reported in the POLR3B subunit only, associated with neurodevelopmental disorder, epilepsy, and peripheral neuropathy.
Luiza L. P. Ramos   +46 more
wiley   +1 more source

Nonviral Gold Nanoparticle‐Mediated Delivery of CRISPR‐Cas9 Ribonucleoprotein and Long DNA Transgenes Into Primary Blood Cells

open access: yesAdvanced NanoBiomed Research, EarlyView.
CRISPR/Cas9 has revolutionized the field of gene therapy, but delivery remains an outstanding issue. We propose a nonviral gold‐nanoparticle platform for co‐delivery of CRISPR/Cas9 ribonucleoprotein and long 2.1 kilobase dsDNA transgene constructs. This CRISPR‐AuNP is inexpensive to produce and mediate gene editing and DNA delivery in T cells and CD34+
Rachel A. Cunningham   +8 more
wiley   +1 more source

Mesoporous Silica Nanoparticles With Customized Drug Ratio/Loading for Effective Treatment of Gemcitabine‐Resistant Pancreatic Tumors

open access: yesAdvanced NanoBiomed Research, EarlyView.
Redox responsive mesoporous silica nanoparticles (MSNs) were engineered to deliver gemcitabine (Gem) and cisplatin (cisPt) at defined ratios to overcome chemoresistance in pancreatic ductal adenocarcinoma (PDAC). Optimized Gem MSNs and Gem cisPt MSNs enhanced cytotoxicity in murine and human Gem resistant models, with select formulations inducing ...
Tamanna Binte Huq   +5 more
wiley   +1 more source

Cadmium inhibits hSMUG1-mediated uracil excision: quantitative analysis and mitigation by <i>Ganoderma lucidum</i> extracts. [PDF]

open access: yesJ Enzyme Inhib Med Chem
Chang HL   +10 more
europepmc   +1 more source

SauCas9-based cell cycle-dependent genome editing via AAV delivery. [PDF]

open access: yesMol Ther Adv
Matsugi E   +5 more
europepmc   +1 more source

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