Literature DB >> 31951831

Encouraging and Unsettling Findings in Long-Term Follow-up of AAV Gene Transfer.

Roland W Herzog1.   

Abstract

Mesh：

Year:  2020        PMID： 31951831      PMCID： PMC7001052          DOI： 10.1016/j.ymthe.2020.01.007

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  6 in total

1.  Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.

Authors:  Albert M Maguire; Stephen Russell; Jennifer A Wellman; Daniel C Chung; Zi-Fan Yu; Amy Tillman; Janet Wittes; Julie Pappas; Okan Elci; Kathleen A Marshall; Sarah McCague; Hannah Reichert; Maria Davis; Francesca Simonelli; Bart P Leroy; J Fraser Wright; Katherine A High; Jean Bennett
Journal:  Ophthalmology       Date:  2019-06-22       Impact factor: 12.079

2.  Liver Gene Therapy: Reliable and Durable?

Authors:  Roland W Herzog; Glenn F Pierce
Journal:  Mol Ther       Date:  2019-10-17       Impact factor: 11.454

3.  The Balance between CD8+ T Cell-Mediated Clearance of AAV-Encoded Antigen in the Liver and Tolerance Is Dependent on the Vector Dose.

Authors:  Sandeep R P Kumar; Brad E Hoffman; Cox Terhorst; Ype P de Jong; Roland W Herzog
Journal:  Mol Ther       Date:  2017-03-09       Impact factor: 11.454

4.  Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A.

Authors:  K John Pasi; Savita Rangarajan; Nina Mitchell; Will Lester; Emily Symington; Bella Madan; Michael Laffan; Chris B Russell; Mingjin Li; Glenn F Pierce; Wing Y Wong
Journal:  N Engl J Med       Date:  2020-01-02       Impact factor: 91.245

5.  5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.

Authors:  Christian Mueller; Gwladys Gernoux; Alisha M Gruntman; Florie Borel; Emer P Reeves; Roberto Calcedo; Farshid N Rouhani; Anthony Yachnis; Margaret Humphries; Martha Campbell-Thompson; Louis Messina; Jeffrey D Chulay; Bruce Trapnell; James M Wilson; Noel G McElvaney; Terence R Flotte
Journal:  Mol Ther       Date:  2017-04-10       Impact factor: 11.454

6.  Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans.

Authors:  Gwladys Gernoux; Alisha M Gruntman; Meghan Blackwood; Marina Zieger; Terence R Flotte; Christian Mueller
Journal:  Mol Ther       Date:  2020-01-13       Impact factor: 11.454
  6 in total
  5 in total

1.  Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency.

Authors:  Chun-Hwei Tai; Ni-Chung Lee; Yin-Hsiu Chien; Barry J Byrne; Shin-Ichi Muramatsu; Sheng-Hong Tseng; Wuh-Liang Hwu
Journal:  Mol Ther       Date:  2021-11-08       Impact factor: 11.454

2.  Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery.

Authors:  Yeon-Suk Yang; Jung-Min Kim; Jun Xie; Sachin Chaugule; Chujiao Lin; Hong Ma; Edward Hsiao; Jaehyoung Hong; Hyonho Chun; Eileen M Shore; Frederick S Kaplan; Guangping Gao; Jae-Hyuck Shim
Journal:  Nat Commun       Date:  2022-10-19       Impact factor: 17.694

Review 3.  Engineering monoclonal antibody-based contraception and multipurpose prevention technologies†.

Authors:  Deborah J Anderson; Joseph A Politch; Richard A Cone; Larry Zeitlin; Samuel K Lai; Philip J Santangelo; Thomas R Moench; Kevin J Whaley
Journal:  Biol Reprod       Date:  2020-08-04       Impact factor: 4.161

4.  AAV9-mediated AIRE gene delivery clears circulating antibodies and tissue T-cell infiltration in a mouse model of autoimmune polyglandular syndrome type-1.

Authors:  Sarah Almaghrabi; Mimoun Azzouz; Rachid Tazi Ahnini
Journal:  Clin Transl Immunology       Date:  2020-09-03

5.  Engineering and In Vitro Selection of a Novel AAV3B Variant with High Hepatocyte Tropism and Reduced Seroreactivity.

Authors:  Moanaro Biswas; Damien Marsic; Ning Li; Chenhui Zou; Gloria Gonzalez-Aseguinolaza; Irene Zolotukhin; Sandeep R P Kumar; Jyoti Rana; John S S Butterfield; Oleksandr Kondratov; Ype P de Jong; Roland W Herzog; Sergei Zolotukhin
Journal:  Mol Ther Methods Clin Dev       Date:  2020-10-04       Impact factor: 6.698
  5 in total

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