而 AAV 载体是体内基因治疗的主要递送方式。 除了有效递送以外，CRISPR 基因编辑疗法在体内应用的第二个挑战在于其潜在的免疫原性，尤其是由于 ...

NanoCas, an ultracompact nuclease, enables efficient muscle editing in NHPs using a single AAV - advancing gene editing for hard-to-target tissues.

引言基因治疗为许多遗传性疾病提供了治愈的希望。肝脏作为人体重要的代谢器官，一直是基因治疗研究的重点之一。肝脏基因治疗不仅可以治疗许多与肝脏相关的遗传性疾病，如血友病和高氨血症等，而且具有极大的临床应用潜力。然而，尽管腺相关病毒（AAV，Adeno-Associated ...

These include the refinement of Ad vectors to improve their safety profile, the discovery of new AAV serotypes, the evolution of lipid nanoparticle (LNP) engineering, the discovery of CRISPR ...

As translational CRISPR-based research continues to make waves in laboratories and the clinic, scientists across life science disciplines pursue out-of-the-ordinary genome editing applications. From ...

Advances in CRISPR technologies promise to accelerate the burgeoning pipeline of gene-editing therapies and broaden access to these disease-altering therapies.

View Full Profile. Learn about our Editorial Policies. “What’s significant about this is it’s taking CRISPR to that next step of what it can be used for, and in this case, it’s correcting mutations ...

Then Crispr came along—the elegant enzymatic apparatus ... As for delivery systems, the family of adeno-associated virus (AAV) vectors already provides the ability to achieve in vivo editing ...

CRISPR is a technology that can be used to edit genes and, as such, will likely change the world. The essence of CRISPR is simple: it’s a way of finding a specific bit of DNA inside a cell.

Researchers have been working to find CRISPR/Cas nucleases with greater specificity ... which they then packaged into AAV viruses. In vivo experiments conducted on mice showed that ChCas12b ...