Conventional adeno-associated viruses (AAVs) are limited by insufficient delivery to the relevant ... attempts are being made to deliver AAV gene therapy directly to the brain parenchyma or ...

Most recently approved gene therapy products use an adeno-associated virus (AAV) as their gene delivery vector. For example, Elevidys, a new treatment for paediatric patients with DMD that was ...

CRISPR gene editing has revolutionized genetics, but delivery challenges have restricted its clinical applications primarily ...

3 Adeno-associated virus (AAV) vectors are used as the delivery carrier for CRISPR gene therapy, with the CRISPR system being packaged as plasmid DNA encoding its components such as Cas9 and guide ...

The same could be said for AAV gene therapy. “The old adage goes that gene therapy is all about delivery, and if you are able to get your therapy to the right tissue or cell type, they will ...

The nano-complex is constructed by the sequential mixing of AAV9, TA, and polymers in an aqueous solution. The nano-complexes released the loaded-AAV intracellularly and exhibited efficient gene ...

In addition, first-generation CRISPR systems (Cas9 and Cas12a) are too large for efficient in vivo delivery ... associated viral (AAV) vectors.” When targeting the PCSK9 gene in mouse liver ...

More recently, Bluebird Bio’s Zynteglo (betibeglogene autotemcel), a cell-based gene therapy ... adeno-associated virus/AAV, capsids, herpes simplex virus/HSV, and virus-like particles/VLPs ...

These systems play a crucial role in gene therapy ... such as CRISPR and synthetic vector development, has enabled more efficient and targeted gene delivery methods, expanding the range of ...

This article tells the story of Victoria Gray, the first patient to receive a CRISPR cell-based gene therapy for sickle cell disease (SCD). We explore the struggles of SCD, the mistreatment of ...

A key to some of the gene therapy approaches for potentially treating RA lies in CRISPR-Cas gene editing ... One challenge is to “multiplex” drug delivery in a way that will get cells that ...

delivery methods and emphasize their advantages for research and gene therapy applications. CRISPR-Cas enzymes offer precision and ease in genome editing; however, they are also associated with ...