[HTML][HTML] 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 …
[HTML][HTML] Engineering the next generation of cell-based therapeutics
Cell-based therapeutics are an emerging modality with the potential to treat many currently
intractable diseases through uniquely powerful modes of action. Despite notable recent …
intractable diseases through uniquely powerful modes of action. Despite notable recent …
[HTML][HTML] 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 …
[HTML][HTML] Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity
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 …
on-target editing efficiency than current adenine base editors (ABEs). To develop a CBE that …
[HTML][HTML] 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 …
Base editing correction of hypertrophic cardiomyopathy in human cardiomyocytes and humanized mice
The most common form of genetic heart disease is hypertrophic cardiomyopathy (HCM),
which is caused by variants in cardiac sarcomeric genes and leads to abnormal heart …
which is caused by variants in cardiac sarcomeric genes and leads to abnormal heart …
[HTML][HTML] Assessing and advancing the safety of CRISPR-Cas tools: from DNA to RNA editing
CRISPR-Cas gene editing has revolutionized experimental molecular biology over the past
decade and holds great promise for the treatment of human genetic diseases. Here we …
decade and holds great promise for the treatment of human genetic diseases. Here we …
Precise plant genome editing using base editors and prime editors
The development of CRISPR–Cas systems has sparked a genome editing revolution in
plant genetics and breeding. These sequence-specific RNA-guided nucleases can induce …
plant genetics and breeding. These sequence-specific RNA-guided nucleases can induce …
In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice
Hutchinson–Gilford progeria syndrome (HGPS or progeria) is typically caused by a
dominant-negative C• G-to-T• A mutation (c. 1824 C> T; p. G608G) in LMNA, the gene that …
dominant-negative C• G-to-T• A mutation (c. 1824 C> T; p. G608G) in LMNA, the gene that …
Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors
The development of new CRISPR–Cas genome editing tools continues to drive major
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …