Harnessing the evolving CRISPR/Cas9 for precision oncology

T Li, S Li, Y Kang, J Zhou, M Yi - Journal of Translational Medicine, 2024 - Springer
Abstract The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9
system, a groundbreaking innovation in genetic engineering, has revolutionized our …

Engineering abiotic stress tolerance in crop plants through CRISPR genome editing

M Rahman, S Zulfiqar, MA Raza, N Ahmad, B Zhang - Cells, 2022 - mdpi.com
Environmental abiotic stresses challenge food security by depressing crop yields often
exceeding 50% of their annual production. Different methods, including conventional as well …

Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy

CRR Alves, LL Ha, R Yaworski, ER Sutton… - Nature biomedical …, 2024 - nature.com
Spinal muscular atrophy (SMA) is caused by mutations in SMN1. SMN2 is a paralogous
gene with a C• G-to-T• A transition in exon 7, which causes this exon to be skipped in most …

Disease modeling by efficient genome editing using a near PAM-less base editor in vivo

M Rosello, M Serafini, L Mignani, D Finazzi… - Nature …, 2022 - nature.com
Base Editors are emerging as an innovative technology to introduce point mutations in
complex genomes. So far, the requirement of an NGG Protospacer Adjacent Motif (PAM) at a …

Cytosine base editors with increased PAM and deaminase motif flexibility for gene editing in zebrafish

Y Zhang, Y Liu, W Qin, S Zheng, J **ao, X **a… - Nature …, 2024 - nature.com
Cytosine base editing is a powerful tool for making precise single nucleotide changes in
cells and model organisms like zebrafish, which are valuable for studying human diseases …

High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease

V Bzhilyanskaya, L Ma, S Liu, LR Fox… - Science translational …, 2024 - science.org
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 (CYBB) gene. The …

Caenorhabditis elegans for research on cancer hallmarks

J Cerón - Disease Models & Mechanisms, 2023 - journals.biologists.com
After decades of research, our knowledge of the complexity of cancer mechanisms,
elegantly summarized as 'hallmarks of cancer', is expanding, as are the therapeutic …

[HTML][HTML] Base editing as a genetic treatment for spinal muscular atrophy

CRR Alves, LL Ha, R Yaworski, CR Lazzarotto… - bioRxiv, 2023 - ncbi.nlm.nih.gov
Spinal muscular atrophy (SMA) is a devastating neuromuscular disease caused by
mutations in the SMN1 gene. Despite the development of various therapies, outcomes can …

Unraveling the mechanisms of PAMless DNA interrogation by SpRY-Cas9

GN Hibshman, JPK Bravo, MM Hooper… - Nature …, 2024 - nature.com
CRISPR-Cas9 is a powerful tool for genome editing, but the strict requirement for an NGG
protospacer-adjacent motif (PAM) sequence immediately next to the DNA target limits the …

Targeting hepatitis B virus DNA using designer gene editors

H Zhang, T Tu - Clinics in Liver Disease, 2023 - liver.theclinics.com
Targeting Hepatitis B Virus DNA Using Designer Gene Editors - Clinics in Liver Disease Skip
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