Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

EA Taha, J Lee, A Hotta - Journal of Controlled Release, 2022 - Elsevier
The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …

CRISPR-based genome editing through the lens of DNA repair

TS Nambiar, L Baudrier, P Billon, A Ciccia - Molecular cell, 2022 - cell.com
Genome editing technologies operate by inducing site-specific DNA perturbations that are
resolved by cellular DNA repair pathways. Products of genome editors include DNA breaks …

Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity

ME Neugebauer, A Hsu, M Arbab, NA Krasnow… - Nature …, 2023 - nature.com
Cytosine base editors (CBEs) are larger and can suffer from higher off-target activity or lower
on-target editing efficiency than current adenine base editors (ABEs). To develop a CBE that …

Re-engineering the adenine deaminase TadA-8e for efficient and specific CRISPR-based cytosine base editing

L Chen, B Zhu, G Ru, H Meng, Y Yan, M Hong… - Nature …, 2023 - nature.com
Cytosine base editors (CBEs) efficiently generate precise C· G-to-T· A base conversions, but
the activation-induced cytidine deaminase/apolipoprotein B mRNA-editing enzyme catalytic …

Engineering a precise adenine base editor with minimal bystander editing

L Chen, S Zhang, N Xue, M Hong, X Zhang… - Nature Chemical …, 2023 - nature.com
Adenine base editors (ABEs) catalyze A-to-G transitions showing broad applications, but
their bystander mutations and off-target editing effects raise safety concerns. Through …

Improved cytosine base editors generated from TadA variants

DK Lam, PR Feliciano, A Arif, T Bohnuud… - Nature …, 2023 - nature.com
Cytosine base editors (CBEs) enable programmable genomic C· G-to-T· A transition
mutations and typically comprise a modified CRISPR–Cas enzyme, a naturally occurring …

In vivo somatic cell base editing and prime editing

GA Newby, DR Liu - Molecular Therapy, 2021 - cell.com
Recent advances in genome editing technologies have magnified the prospect of single-
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …

High-purity production and precise editing of DNA base editing ribonucleoproteins

HK Jang, DH Jo, SN Lee, CS Cho, YK Jeong… - Science …, 2021 - science.org
Ribonucleoprotein (RNP) complex–mediated base editing is expected to be greatly
beneficial because of its reduced off-target effects compared to plasmid-or viral vector …

Base editing of organellar DNA with programmable deaminases

JS Kim, J Chen - Nature Reviews Molecular Cell Biology, 2024 - nature.com
Mitochondria and chloroplasts are organelles that include their own genomes, which
encode key genes for ATP production and carbon dioxide fixation, respectively. Mutations in …

In the business of base editors: evolution from bench to bedside

EM Porto, AC Komor - PLoS biology, 2023 - journals.plos.org
With the advent of recombinant DNA technology in the 1970s, the idea of using gene
therapies to treat human genetic diseases captured the interest and imagination of scientists …