Literature DB >> 33495615

The clinical landscape for AAV gene therapies.

Dmitry A Kuzmin, Maria V Shutova, Natalie R Johnston, Owen P Smith, Vasily V Fedorin, Yury S Kukushkin, Johannes C M van der Loo, Elaine C Johnstone.   

Abstract

Year:  2021        PMID： 33495615     DOI： 10.1038/d41573-021-00017-7

Source DB:  PubMed          Journal:  Nat Rev Drug Discov        ISSN： 1474-1776            Impact factor:   84.694


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  61 in total

1.  AAV-BR1 targets endothelial cells in the retina to reveal their morphological diversity and to deliver Cx43.

Authors:  Elena Ivanova; Carlo Corona; Cyril G Eleftheriou; Randy F Stout; Jakob Körbelin; Botir T Sagdullaev
Journal:  J Comp Neurol       Date:  2021-12-09       Impact factor: 3.215

Review 2.  Nuclear-Import Receptors Counter Deleterious Phase Transitions in Neurodegenerative Disease.

Authors:  Hana M Odeh; Charlotte M Fare; James Shorter
Journal:  J Mol Biol       Date:  2021-08-28       Impact factor: 5.469

3.  Rescue of a familial dysautonomia mouse model by AAV9-Exon-specific U1 snRNA.

Authors:  Giulia Romano; Federico Riccardi; Erica Bussani; Simone Vodret; Danilo Licastro; Isabella Ragone; Giuseppe Ronzitti; Elisabetta Morini; Susan A Slaugenhaupt; Franco Pagani
Journal:  Am J Hum Genet       Date:  2022-07-28       Impact factor: 11.043

4.  Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors.

Authors:  Jessie R Davis; Xiao Wang; Isaac P Witte; Tony P Huang; Jonathan M Levy; Aditya Raguram; Samagya Banskota; Nabil G Seidah; Kiran Musunuru; David R Liu
Journal:  Nat Biomed Eng       Date:  2022-07-28       Impact factor: 29.234

Review 5.  Therapeutic in vivo delivery of gene editing agents.

Authors:  Aditya Raguram; Samagya Banskota; David R Liu
Journal:  Cell       Date:  2022-07-06       Impact factor: 66.850

6.  Distribution of cardiomyocyte-selective adeno-associated virus serotype 9 vectors in swine following intracoronary and intravenous infusion.

Authors:  Jinliang Li; Shannon C Kelly; Jan R Ivey; Pamela K Thorne; Kelly P Yamada; Tadao Aikawa; Renata Mazurek; James R Turk; Kleiton Augusto Santos Silva; Amira R Amin; Darla L Tharp; Christina M Mueller; Hrishikesh Thakur; Emily V Leary; Timothy L Domeier; R Scott Rector; Kenneth Fish; Federico Cividini; Kiyotake Ishikawa; Craig A Emter; Michael S Kapiloff
Journal:  Physiol Genomics       Date:  2022-06-01       Impact factor: 4.297

Review 7.  Evaluating the state of the science for adeno-associated virus integration: An integrated perspective.

Authors:  Denise E Sabatino; Frederic D Bushman; Randy J Chandler; Ronald G Crystal; Beverly L Davidson; Ricardo Dolmetsch; Kevin C Eggan; Guangping Gao; Irene Gil-Farina; Mark A Kay; Douglas M McCarty; Eugenio Montini; Adora Ndu; Jing Yuan
Journal:  Mol Ther       Date:  2022-06-10       Impact factor: 12.910

8.  Adeno-associated virus mediated expression of monoclonal antibody MR191 protects mice against Marburg virus and provides long-term expression in sheep.

Authors:  Amira D Rghei; Laura P van Lieshout; Wenguang Cao; Shihua He; Kevin Tierney; Jordyn A Lopes; Nicole Zielinska; Enzo M Baracuhy; Elena S B Campbell; Jessica A Minott; Matthew M Guilleman; Pamela C Hasson; Brad Thompson; Khalil Karimi; Byram W Bridle; Leonardo Susta; Xiangguo Qiu; Logan Banadyga; Sarah K Wootton
Journal:  Gene Ther       Date:  2022-09-01       Impact factor: 4.184

Review 9.  In vivo somatic cell base editing and prime editing.

Authors:  Gregory A Newby; David R Liu
Journal:  Mol Ther       Date:  2021-09-10       Impact factor: 11.454

10.  Short regulatory DNA sequences to target brain endothelial cells for gene therapy.

Authors:  Hanna Graßhoff; Helge Müller-Fielitz; Godwin K Dogbevia; Jakob Körbelin; Jacqueline Bannach; Carl Mg Vahldieck; Kristina Kusche-Vihrog; Olaf Jöhren; Oliver J Müller; Ruben Nogueiras; Vincent Prevot; Markus Schwaninger
Journal:  J Cereb Blood Flow Metab       Date:  2021-08-24       Impact factor: 6.960
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