Computational tools and resources for CRISPR/Cas genome editing

C Li, W Chu, RA Gill, S Sang, Y Shi, X Hu… - Genomics …, 2023 - academic.oup.com
The past decade has witnessed a rapid evolution in identifying more versatile clustered
regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) …

Design and analysis of CRISPR–Cas experiments

RE Hanna, JG Doench - Nature biotechnology, 2020 - nature.com
A large and ever-expanding set of CRISPR–Cas systems now enables the rapid and flexible
manipulation of genomes in both targeted and large-scale experiments. Numerous software …

Potent and uniform fetal hemoglobin induction via base editing

T Mayuranathan, GA Newby, R Feng, Y Yao… - Nature …, 2023 - nature.com
Inducing fetal hemoglobin (HbF) in red blood cells can alleviate β-thalassemia and sickle
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …

Mammalian retrovirus-like protein PEG10 packages its own mRNA and can be pseudotyped for mRNA delivery

M Segel, B Lash, J Song, A Ladha, CC Liu, X Jin… - Science, 2021 - science.org
Eukaryotic genomes contain domesticated genes from integrating viruses and mobile
genetic elements. Among these are homologs of the capsid protein (known as Gag) of long …

Deleting DNMT3A in CAR T cells prevents exhaustion and enhances antitumor activity

B Prinzing, CC Zebley, CT Petersen, Y Fan… - Science translational …, 2021 - science.org
Chimeric antigen receptor (CAR) T cell therapy is revolutionizing cancer immunotherapy for
patients with B cell malignancies and is now being developed for solid tumors and chronic …

Base editing of haematopoietic stem cells rescues sickle cell disease in mice

GA Newby, JS Yen, KJ Woodard, T Mayuranathan… - Nature, 2021 - nature.com
Sickle cell disease (SCD) is caused by a mutation in the β-globin gene HBB. We used a
custom adenine base editor (ABE8e-NRCH), to convert the SCD allele (HBB S) into …

Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing

ML Leibowitz, S Papathanasiou, PA Doerfler… - Nature …, 2021 - nature.com
Genome editing has therapeutic potential for treating genetic diseases and cancer.
However, the currently most practicable approaches rely on the generation of DNA double …

cBAF complex components and MYC cooperate early in CD8+ T cell fate

A Guo, H Huang, Z Zhu, MJ Chen, H Shi, S Yuan… - Nature, 2022 - nature.com
The identification of mechanisms to promote memory T (Tmem) cells has important
implications for vaccination and anti-cancer immunotherapy,,–. Using a CRISPR-based …

Base-resolution models of transcription-factor binding reveal soft motif syntax

Ž Avsec, M Weilert, A Shrikumar, S Krueger… - Nature …, 2021 - nature.com
The arrangement (syntax) of transcription factor (TF) binding motifs is an important part of the
cis-regulatory code, yet remains elusive. We introduce a deep learning model, BPNet, that …

[HTML][HTML] Single-cell CRISPR screens in vivo map T cell fate regulomes in cancer

P Zhou, H Shi, H Huang, X Sun, S Yuan, NM Chapman… - Nature, 2023 - nature.com
CD8+ cytotoxic T cells (CTLs) orchestrate antitumour immunity and exhibit inherent
heterogeneity,, with precursor exhausted T (Tpex) cells but not terminally exhausted T (Tex) …