Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9 [PDF]
Curiel, David T +5 more
core +1 more source
Biomaterial-Based CRISPR/Cas9 Delivery Systems for Tumor Treatment
Mengmeng Li +8 more
openalex +1 more source
Otimização de uma plataforma baseada na edição gênica por CRISPR/Cas9 para a produção de células T alogênicas expressando o receptor quimérico de antígeno (CAR) [PDF]
Sarah Caroline Gomes de Lima
openalex +1 more source
Mutant p53 drives oncogenic splicing to promote the progression of ovarian cancer by partnering with the spliceosome factor SNRPD2. Therefore, it is engineered iRGD‐exosomes to co‐deliver siRNAs against both targets. This approach restored tumor‐suppressive mRNA isoforms, effectively enhanced sensitivity to cisplatin, and ultimately blocked tumor ...
Wei Zhao +14 more
wiley +1 more source
Multiplex CRISPR/Cas9-mediated editing of seven glycosyltransferase homologs in <i>Nicotiana benthamiana</i> to produce stable, Cas9-free, glycoengineered plants. [PDF]
Kaur C +9 more
europepmc +1 more source
Discovery of a Novel DNMT1 Inhibitor with Improved Efficacy in Treating β‐Thalassemia
Context of Research: β‐thalassemia affects millions worldwide. DNMT inhibitors are effective HbF‐inducers that benefit patients with β‐thalassemia. Existing DNMT inhibitors are not approved for β‐thalassemia treatment due to dose‐limiting toxicity.What We Find: DMT207 traps DNMT1 into helix‐kinked inactive conformation and enhances its interaction with
Yijie Shen +19 more
wiley +1 more source
CRISPR-Cas systems against carbapenem resistance: from proof-of-concept to clinical translation. [PDF]
Tsolakidou PJ.
europepmc +1 more source
Folic acid (FA) and multivitamins/minerals (MVM) are recommended for the prevention of neural tube defects (NTDs). Using a novel human neural tube‐like organoid system and mouse models, the study compares the effectiveness of two supplements on NTDs and cellular behaviors.
Huili Li +3 more
wiley +1 more source
Stage- and tissue-specific gene editing using 4-OHT-inducible Cas9 in whole organism. [PDF]
Li Y +9 more
europepmc +1 more source

