delivery technologies that have been used to enable therapeutic in vivo gene editing,including viral vectors,lipid nanoparticles,and virus-like particles.Since no single delivery modality is likely to be appropriate for every possible application,we compare the benefits and drawbacks of each method and...
Current state-of-the-art delivery systems, including viral vectors, LNPs, and VLPs, can satisfy these key criteria for efficient in vivo delivery vehicles and are therefore well suited for the in vivo delivery of gene editing agents. Viral delivery Viruses naturally evolved to overcome barriers ...
基因编辑大神David Liu在Cell上又祭出一篇review文章《Therapeutic in vivo delivery of gene editing agents》,对这一治疗技术的核心瓶颈递送环节做了系统的梳理和评价。网页链接(22)00395-6 1. 基因编辑治疗策略 目前已经有几种体内基因编辑疗法,包括核酸酶(当然就有CRISPR-Cas)、Base Editor和Prime Editor等,具体...
Amaurosis 10, as well as CRISPR/Cas9-edited T cells designed to target and destroy cancer cells. Further advances in therapeutic genome editing will rely on a safe and more efficient method ofin vivoCRISPR/Cas9 delivery and improved efficiency of homology-directed repair for site-specific gene in...
Over the past few years, clinical translation of bench research resulted in the first examples of therapeutic success for the field of gene transfer. Adeno-associated virus (AAV) vectors are among the most suitable tools for in vivo gene delivery given their superior efficiency of transduction of...
As around half of known pathogenic genetic variants are due to SNVs, base editing holds great potential for the treatment of numerous genetic diseases, through either temporary RNA or permanent DNA base alterations. Recent advances in the specificity, efficiency, precision and delivery of DNA and ...
Methods to deliver gene editing agents in vivo as ribonucleoproteins could offer safety advantages over nucleic acid delivery approaches. We report the development and application of engineered DNA-free virus-like particles (eVLPs) that efficiently package and deliver base editor or Cas9 ribonucleoproteins...
However, there are still limitations hindering an ideal application of the system: inefficient delivery, dysregulation of the delivered gene, the immune response against the CRISPR system, the off-target effects or the unintended on-target mutations. In addition, there are recent discoveries that ...
Delivery technologies that have serendipitously been developed over the past couple decades in the protein and nucleic acid delivery fields have been crucial to genome editing success to date, including adeno-associated viral and lentiviral vectors for gene therapy and lipid nanoparticle and other non-...
Technical barriers to mRNA vaccines are centered on their molecular design and in vivo delivery efficiency. mRNA modification and the sequence design of the its regulatory and coding regions play a crucial role in determining mRNA stability and translation efficiency. The antigen translation efficiency ...