Engineering the next-generation of CAR T-cells with CRISPR-Cas9 gene editing
A Dimitri, F Herbst, JA Fraietta - Molecular cancer, 2022 - Springer
Abstract Chimeric Antigen Receptor (CAR) T-cells represent a breakthrough in personalized
cancer therapy. In this strategy, synthetic receptors comprised of antigen recognition …
cancer therapy. In this strategy, synthetic receptors comprised of antigen recognition …
CRISPR-based genome editing through the lens of DNA repair
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 …
resolved by cellular DNA repair pathways. Products of genome editors include DNA breaks …
Engineered pegRNAs improve prime editing efficiency
JW Nelson, PB Randolph, SP Shen, KA Everette… - Nature …, 2022 - nature.com
Prime editing enables the installation of virtually any combination of point mutations, small
insertions or small deletions in the DNA of living cells. A prime editing guide RNA (pegRNA) …
insertions or small deletions in the DNA of living cells. A prime editing guide RNA (pegRNA) …
Potent and uniform fetal hemoglobin induction via base editing
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 …
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …
Engineered miniature CRISPR-Cas system for mammalian genome regulation and editing
Compact and versatile CRISPR-Cas systems will enable genome engineering applications
through high-efficiency delivery in a wide variety of contexts. Here, we create an efficient …
through high-efficiency delivery in a wide variety of contexts. Here, we create an efficient …
Base editing of haematopoietic stem cells rescues sickle cell disease in mice
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 …
custom adenine base editor (ABE8e-NRCH), to convert the SCD allele (HBB S) into …
High-throughput continuous evolution of compact Cas9 variants targeting single-nucleotide-pyrimidine PAMs
TP Huang, ZJ Heins, SM Miller, BG Wong… - Nature …, 2023 - nature.com
Despite the availability of Cas9 variants with varied protospacer-adjacent motif (PAM)
compatibilities, some genomic loci—especially those with pyrimidine-rich PAM sequences …
compatibilities, some genomic loci—especially those with pyrimidine-rich PAM sequences …
In vivo somatic cell base editing and prime editing
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 …
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …
Compact zinc finger base editors that edit mitochondrial or nuclear DNA in vitro and in vivo
JCW Willis, P Silva-Pinheiro, L Widdup… - Nature …, 2022 - nature.com
DddA-derived cytosine base editors (DdCBEs) use programmable DNA-binding TALE
repeat arrays, rather than CRISPR proteins, a split double-stranded DNA cytidine …
repeat arrays, rather than CRISPR proteins, a split double-stranded DNA cytidine …
Multiplex base-and prime-editing with drive-and-process CRISPR arrays
Q Yuan, X Gao - Nature communications, 2022 - nature.com
Current base-and prime-editing technologies lack efficient strategies to edit multiple
genomic loci simultaneously, limiting their applications in complex genomics and polygenic …
genomic loci simultaneously, limiting their applications in complex genomics and polygenic …