Asset Details
Do you wish to reserve the book?
The delivery challenge: fulfilling the promise of therapeutic genome editing
Hey, we have placed the reservation for you!
By the way, why not check out events that you can attend while you pick your title.
Oops! Something went wrong.
Looks like we were not able to place the reservation. Kindly try again later.
Are you sure you want to remove the book from the shelf?
The delivery challenge: fulfilling the promise of therapeutic genome editing
Do you wish to request the book?
The delivery challenge: fulfilling the promise of therapeutic genome editing
Please be aware that the book you have requested cannot be checked out. If you would like to checkout this book, you can reserve another copy
We have requested the book for you!
Your request is successful and it will be processed during the Library working hours. Please check the status of your request in My Requests.
Oops! Something went wrong.
Looks like we were not able to place your request. Kindly try again later.
Journal Article
The delivery challenge: fulfilling the promise of therapeutic genome editing
2020
Overview
Genome editing has the potential to treat an extensive range of incurable monogenic and complex diseases. In particular, advances in sequence-specific nuclease technologies have dramatically accelerated the development of therapeutic genome editing strategies that are based on either the knockout of disease-causing genes or the repair of endogenous mutated genes. These technologies are progressing into human clinical trials. However, challenges remain before the therapeutic potential of genome editing can be fully realized. 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-viral vectors for nucleic acid and protein delivery. However, the efficiency and tissue targeting capabilities of these vehicles must be further improved. In addition, the genome editing enzymes themselves need to be optimized, and challenges regarding their editing efficiency, specificity and immunogenicity must be addressed. Emerging protein engineering and synthetic chemistry approaches can offer solutions and enable the development of safe and efficacious clinical genome editing.
As CRISPR therapies move into clinical testing, David Schaffer and colleagues review a raft of different delivery technologies being road tested to address cargo capacity limitations, maximize potency, minimize off-target effects and avoid immunogenicity.
Publisher
Nature Publishing Group US,Nature Publishing Group
Subject
/ Biomedical and Life Sciences
/ Biomedical Engineering/Biotechnology
/ CRISPR
/ CRISPR-Cas Systems - genetics
/ Editing
/ Genes
/ Genetic Diseases, Inborn - genetics
/ Genetic Diseases, Inborn - therapy
/ Genetic Vectors - therapeutic use
/ Genomes
/ Humans
/ Lipids
/ Methods
/ Nanoparticles - therapeutic use
/ Nuclease
/ Proteins
