The promise and challenge of therapeutic genome editing
JA Doudna - Nature, 2020 - nature.com
Genome editing, which involves the precise manipulation of cellular DNA sequences to alter
cell fates and organism traits, has the potential to both improve our understanding of human …
cell fates and organism traits, has the potential to both improve our understanding of human …
[HTML][HTML] Improving clinical trial outcomes in amyotrophic lateral sclerosis
Individuals who are diagnosed with amyotrophic lateral sclerosis (ALS) today face the same
historically intransigent problem that has existed since the initial description of the disease in …
historically intransigent problem that has existed since the initial description of the disease in …
Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing
CRISPR–Cas gene editing and messenger RNA-based protein replacement therapy hold
tremendous potential to effectively treat disease-causing mutations with diverse cellular …
tremendous potential to effectively treat disease-causing mutations with diverse cellular …
Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing
Genome editing has therapeutic potential for treating genetic diseases and cancer.
However, the currently most practicable approaches rely on the generation of DNA double …
However, the currently most practicable approaches rely on the generation of DNA double …
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 …
Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system
Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by
mutations in the dystrophin gene (DMD). Previously, we applied CRISPR-Cas9–mediated …
mutations in the dystrophin gene (DMD). Previously, we applied CRISPR-Cas9–mediated …
The next generation of CRISPR–Cas technologies and applications
A Pickar-Oliver, CA Gersbach - Nature reviews Molecular cell biology, 2019 - nature.com
The prokaryote-derived CRISPR–Cas genome editing systems have transformed our ability
to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells …
to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells …
Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing
F Chemello, AC Chai, H Li, C Rodriguez-Caycedo… - Science …, 2021 - science.org
Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of
dystrophin, which maintains muscle membrane integrity. We used an adenine base editor …
dystrophin, which maintains muscle membrane integrity. We used an adenine base editor …
Adeno-associated virus vector as a platform for gene therapy delivery
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …
treatment of a variety of human diseases. Recent advances in developing clinically …
[PDF][PDF] 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 …