Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase Complex

Autor: Feijie Zhang, Matthew Tiffany, Ruhikanta A. Meetei, M. Kyle Cromer, Karim Majzoub, Mara Pavel-Dinu, Sirika Pillay, Adam Sheikali, Jan E. Carette, Mark A. Kay, Katja Pekrun, Francesco Puzzo, Hagoon Jang, Gustavo de Alencastro, Matthew H. Porteus

Publikováno v: Mol Ther

Recombinant adeno-associated virus (rAAV) vectors have the unique property of being able to perform genomic targeted integration (TI) without inducing a double-strand break (DSB). In order to improve our understanding of the mechanism behind TI media

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::35065181d1261c0a3966f7b32e3ee768
https://europepmc.org/articles/PMC7934449/

Tracking Adeno-Associated Virus Capsid Evolution by High-Throughput Sequencing

Autor: Mark A. Kay, Jianpeng Xu, Gustavo de Alencastro, Katja Pekrun, Paul N. Valdmanis, Matthew Tiffany

Publikováno v: Hum Gene Ther

Despite early successes using recombinant adeno-associated virus (rAAV) vectors in clinical gene therapy trials, limitations remain making additional advancements a necessity. Some of the challenges include variable levels of pre-existing neutralizin

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c1034075e33d4e1c8ac18d6fdf1771c4
https://europepmc.org/articles/PMC7232707/

Using a barcoded AAV capsid library to select for novel clinically relevant gene therapy vectors

Autor: Sean Nygaard, Ren Song, Feijie Zhang, Jun Liu, Qing-Jun Luo, Mark A. Kay, Matthias Hebrok, Jianpeng Xu, Markus Grompe, Youngjin Kim, Matthew Tiffany, Katja Pekrun, Gustavo de Alencastro, Feorillo Galivo

While gene transfer using recombinant adeno-associated viral (rAAV) vectors have shown success in some clinical trials, there remain many tissues that are not well transduced. Because of the recent success in reprogramming islet derived cells into fu

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7a06e6370b178e16a228e55f7e0983af
https://doi.org/10.1101/683672

Selective targeting of engineered T cells using orthogonal IL-2 cytokine-receptor complexes

Autor: Indigo Chris King, Akanksha Chhabra, David Baker, Leah V. Sibener, Matthew Tiffany, Antoni Ribas, Alan C. Le, Jeffrey A. Bluestone, Leon Su, Stephanie L. Silveria, Benson M. George, Eleonora Trotta, Jonathan T. Sockolosky, Giulia Parisi, Kevin Jude, Judith A. Shizuru, K. Christopher Garcia, Lora Picton

Publikováno v: Science (New York, N.Y.), vol 359, iss 6379
Sockolosky, JT; Trotta, E; Parisi, G; Picton, L; Su, LL; Le, AC; et al.(2018). Selective targeting of engineered T cells using orthogonal IL-2 cytokine-receptor complexes. SCIENCE, 359(6379), 1037-+. doi: 10.1126/science.aar3246. UCLA: Retrieved from: http://www.escholarship.org/uc/item/1fn5n8p9

Engineering cytokine-receptor pairs Interleukin-2 (IL-2) is an important cytokine that helps T cells destroy tumors and virus-infected cells. IL-2 has great therapeutic promise but is limited by toxic side effects and its capacity to both activate an

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::89f7612e9b2dc674d79cf5a181507638
https://escholarship.org/uc/item/1fn5n8p9

253. Expanded Packaging Capacity of AAV by Lumenal Charge Alteration

Autor: Mark A. Kay, Matthew Tiffany

Publikováno v: Molecular Therapy. 24:S99-S100

Adeno-associated virus (AAV) is widely believed to be the safest viral vector for gene therapy. Moreover, the array of AAV serotypes available allows transduction of different tissues in vivo. However, one major limitation of AAV is the relatively sm

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::17c18c6cb8cba6a89fe3305e1585485e
https://doi.org/10.1016/s1525-0016(16)33062-3