CRISPR gene therapy: applications, limitations, and implications for the future
A series of recent discoveries harnessing the adaptive immune system of prokaryotes to
perform targeted genome editing is having a transformative influence across the biological …
perform targeted genome editing is having a transformative influence across the biological …
CRISPR-based therapeutic genome editing: strategies and in vivo delivery by AAV vectors
The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …
Control of the activity of CAR-T cells within tumours via focused ultrasound
Focused ultrasound can deliver energy safely and non-invasively into tissues at depths of
centimetres. Here we show that the genetics and cellular functions of chimeric antigen …
centimetres. Here we show that the genetics and cellular functions of chimeric antigen …
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models
CH Hakim, SRP Kumar, DO Pérez-López… - Nature …, 2021 - nature.com
Abstract Adeno-associated virus (AAV)-mediated CRISPR-Cas9 editing holds promise to
treat many diseases. The immune response to bacterial-derived Cas9 has been speculated …
treat many diseases. The immune response to bacterial-derived Cas9 has been speculated …
AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice
Gene therapy is a potentially curative medicine for many currently untreatable diseases, and
recombinant adeno-associated virus (rAAV) is the most successful gene delivery vehicle for …
recombinant adeno-associated virus (rAAV) is the most successful gene delivery vehicle for …
Photoacoustic molecular imaging-escorted adipose photodynamic–browning synergy for fighting obesity with virus-like complexes
R Chen, S Huang, T Lin, H Ma, W Shan, F Duan… - Nature …, 2021 - nature.com
Photodynamic therapy and adipose browning induction are two promising approaches to
reverse obesity. The former strategy acts rapidly and locally, whereas the latter has a more …
reverse obesity. The former strategy acts rapidly and locally, whereas the latter has a more …
Microneedle-assisted genome editing: A transdermal strategy of targeting NLRP3 by CRISPR-Cas9 for synergistic therapy of inflammatory skin disorders
T Wan, Q Pan, Y Ping - Science advances, 2021 - science.org
We report a dissolvable microneedle (MN) patch that can mediate transdermal codelivery of
CRISPR-Cas9–based genome-editing agents and glucocorticoids for the effective treatment …
CRISPR-Cas9–based genome-editing agents and glucocorticoids for the effective treatment …
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 …
AAV-CRISPR gene editing is negated by pre-existing immunity to Cas9
Adeno-associated viral (AAV) vectors are a leading candidate for the delivery of CRISPR-
Cas9 for therapeutic genome editing in vivo. However, AAV-based delivery involves …
Cas9 for therapeutic genome editing in vivo. However, AAV-based delivery involves …
RNA editing: Expanding the potential of RNA therapeutics
RNA therapeutics have had a tremendous impact on medicine, recently exemplified by the
rapid development and deployment of mRNA vaccines to combat the COVID-19 pandemic …
rapid development and deployment of mRNA vaccines to combat the COVID-19 pandemic …