Results 251 to 260 of about 278,076 (329)

Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9 [PDF]

open access: yes, 2019
Curiel, David T   +5 more
core   +1 more source

Biomaterial-Based CRISPR/Cas9 Delivery Systems for Tumor Treatment

open access: gold
Mengmeng Li   +8 more
openalex   +1 more source

Dual Targeting of Mutant p53 and SNRPD2 via Engineered Exosomes Modulates Alternative Splicing to Suppress Ovarian Cancer

open access: yesAdvanced Science, EarlyView.
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

Discovery of a Novel DNMT1 Inhibitor with Improved Efficacy in Treating β‐Thalassemia

open access: yesAdvanced Science, EarlyView.
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

Effectiveness of Multivitamins vs Folic Acid on Prevention of Neural Tube Defects in Mouse Genetic Models and Human Organoids

open access: yesAdvanced Science, EarlyView.
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]

open access: yesJ Cell Biol
Li Y   +9 more
europepmc   +1 more source

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