CRISPR/Cas9 therapeutics: progress and prospects
T Li, Y Yang, H Qi, W Cui, L Zhang, X Fu, X He… - … and Targeted Therapy, 2023 - nature.com
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …
Therapeutic in vivo delivery of gene editing agents
In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins
Methods to deliver gene editing agents in vivo as ribonucleoproteins could offer safety
advantages over nucleic acid delivery approaches. We report the development and …
advantages over nucleic acid delivery approaches. We report the development and …
In vivo hematopoietic stem cell modification by mRNA delivery
L Breda, TE Papp, MP Triebwasser, A Yadegari… - Science, 2023 - science.org
Hematopoietic stem cells (HSCs) are the source of all blood cells over an individual's
lifetime. Diseased HSCs can be replaced with gene-engineered or healthy HSCs through …
lifetime. Diseased HSCs can be replaced with gene-engineered or healthy HSCs through …
Current applications and future perspective of CRISPR/Cas9 gene editing in cancer
SW Wang, C Gao, YM Zheng, L Yi, JC Lu, XY Huang… - Molecular cancer, 2022 - Springer
Clustered regularly interspaced short palindromic repeats (CRISPR) system provides
adaptive immunity against plasmids and phages in prokaryotes. This system inspires the …
adaptive immunity against plasmids and phages in prokaryotes. This system inspires the …
Genotoxic effects of base and prime editing in human hematopoietic stem cells
Base and prime editors (BEs and PEs) may provide more precise genetic engineering than
nuclease-based approaches because they bypass the dependence on DNA double-strand …
nuclease-based approaches because they bypass the dependence on DNA double-strand …
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 …
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 …
their bystander mutations and off-target editing effects raise safety concerns. Through …
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice
Sickle-cell disease (SCD) is caused by an A· T-to-T· A transversion mutation in the β-globin
gene (HBB). Here we show that prime editing can correct the SCD allele (HBB S) to wild …
gene (HBB). Here we show that prime editing can correct the SCD allele (HBB S) to wild …
Human genetic diversity alters off-target outcomes of therapeutic gene editing
CRISPR gene editing holds great promise to modify DNA sequences in somatic cells to treat
disease. However, standard computational and biochemical methods to predict off-target …
disease. However, standard computational and biochemical methods to predict off-target …