Healthy Living: September 10, 2019

Published: Sep. 10, 2019 at 3:38 PM EDT
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The risks, benefits, and future of this groundbreaking technology.


1. Length of efficacy

2. Potential immune response

3. Off-target effects


1. Reduced clinic visits

2. Durability

3. Greater efficacy


1. Regenxbio RGX-314

2. Adverum Biotechnologies

Kiss S, Nieves J, Grishanin R, et al. Ocular safety of long-term suppression of VEGF by intravitreally administered gene therapy, ADVM-022, in non-human primates. Paper presented at: American Society of Gene & Cell Therapy 22nd Annual Meeting; April 29-May 2, 2019; Washington, DC. Accessed June 8, 2019.

Jo DH, Koo T, Cho CS, Kim JH, Kim JS, Kim JH. Long-term effects of in vivo genome editing in the mouse retina using campylobacter jejuni Cas9 expressed via adeno-associated virus [Notes:published online October 17, 2018] . Mol Ther.2019;27(1):130-136. doi:10.1016/j.ymthe.2018.10.009

Cho GY, Schaefer KA, Bassuk AG, Tsang SH, Mahajan VB. Crispr genome surgery in the retina in light of off-targeting. Retina. 2018;38(8):1443-1455. doi:10.1097/IAE.0000000000002197

Reid CA, Nettesheim ER, Connor TB, Lipinski DM. Development of an inducible anti-VEGF rAAV gene therapy strategy for the treatment of wet AMD. Sci Rep. 2018;8(1):11763. doi:10.1038/s41598-018-29726-7

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