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 …
CRISPR technologies for genome, epigenome and transcriptome editing
Our ability to edit genomes lags behind our capacity to sequence them, but the growing
understanding of CRISPR biology and its application to genome, epigenome and …
understanding of CRISPR biology and its application to genome, epigenome and …
Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors
The viral delivery of base editors has been complicated by their size and by the limited
packaging capacity of adeno-associated viruses (AAVs). Typically, dual-AAV approaches …
packaging capacity of adeno-associated viruses (AAVs). Typically, dual-AAV approaches …
Programmed genome editing by a miniature CRISPR-Cas12f nuclease
Z Wu, Y Zhang, H Yu, D Pan, Y Wang, Y Wang… - Nature chemical …, 2021 - nature.com
The RNA-guided CRISPR-associated (Cas) nucleases are versatile tools for genome editing
in various organisms. The large sizes of the commonly used Cas9 and Cas12a nucleases …
in various organisms. The large sizes of the commonly used Cas9 and Cas12a nucleases …
Precise genomic editing of pathogenic mutations in RBM20 rescues dilated cardiomyopathy
Mutations in RNA binding motif protein 20 (RBM20) are a common cause of familial dilated
cardiomyopathy (DCM). Many RBM20 mutations cluster within an arginine/serine-rich (RS …
cardiomyopathy (DCM). Many RBM20 mutations cluster within an arginine/serine-rich (RS …
A highly specific CRISPR-Cas12j nuclease enables allele-specific genome editing
Y Wang, T Qi, J Liu, Y Yang, Z Wang, Y Wang… - Science …, 2023 - science.org
The CRISPR-Cas system can treat autosomal dominant diseases by nonhomologous end
joining (NHEJ) gene disruption of mutant alleles. However, many single-nucleotide …
joining (NHEJ) gene disruption of mutant alleles. However, many single-nucleotide …
Precision genome editing using cytosine and adenine base editors in mammalian cells
Genome editing has transformed the life sciences and has exciting prospects for use in
treating genetic diseases. Our laboratory developed base editing to enable precise and …
treating genetic diseases. Our laboratory developed base editing to enable precise and …
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo
Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic
genome editing but are severely constrained by cargo limits. Simultaneous delivery of …
genome editing but are severely constrained by cargo limits. Simultaneous delivery of …
The NIH somatic cell genome editing program
The move from reading to writing the human genome offers new opportunities to improve
human health. The United States National Institutes of Health (NIH) Somatic Cell Genome …
human health. The United States National Institutes of Health (NIH) Somatic Cell Genome …
Discrimination of single-point mutations in unamplified genomic DNA via Cas9 immobilized on a graphene field-effect transistor
S Balderston, JJ Taulbee, E Celaya, K Fung… - Nature Biomedical …, 2021 - nature.com
Simple and fast methods for the detection of target genes with single-nucleotide specificity
could open up genetic research and diagnostics beyond laboratory settings. We recently …
could open up genetic research and diagnostics beyond laboratory settings. We recently …