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Literature DB >> 34758292

Eliminating Panglossian thinking in development of AAV therapeutics.

Radoslaw Kaczmarek¹, Glenn F Pierce², Declan Noone³, Brian O'Mahony⁴, David Page⁵, Mark W Skinner⁶.

Abstract

Entities: Chemical

Mesh：

Year: 2021 PMID： 34758292 PMCID： PMC8636166 DOI： 10.1016/j.ymthe.2021.10.025

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

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13 in total

1. Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1.

Authors: Amy G Feldman; Julie A Parsons; Cullen M Dutmer; Aravindhan Veerapandiyan; Einar Hafberg; Nolan Maloney; Cara L Mack
Journal: J Pediatr Date: 2020-05-28 Impact factor: 4.406

2. MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates.

Authors: Juliette Hordeaux; Elizabeth L Buza; Brianne Jeffrey; Chunjuan Song; Tahsin Jahan; Yuan Yuan; Yanqing Zhu; Peter Bell; Mingyao Li; Jessica A Chichester; Roberto Calcedo; James M Wilson
Journal: Sci Transl Med Date: 2020-11-11 Impact factor: 17.956

3. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.

Authors: Catherine S Manno; Glenn F Pierce; Valder R Arruda; Bertil Glader; Margaret Ragni; John J Rasko; John Rasko; Margareth C Ozelo; Keith Hoots; Philip Blatt; Barbara Konkle; Michael Dake; Robin Kaye; Mahmood Razavi; Albert Zajko; James Zehnder; Pradip K Rustagi; Hiroyuki Nakai; Amy Chew; Debra Leonard; J Fraser Wright; Ruth R Lessard; Jürg M Sommer; Michael Tigges; Denise Sabatino; Alvin Luk; Haiyan Jiang; Federico Mingozzi; Linda Couto; Hildegund C Ertl; Katherine A High; Mark A Kay
Journal: Nat Med Date: 2006-02-12 Impact factor: 53.440

4. Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy.

Authors: Deepa Chand; Franziska Mohr; Hugh McMillan; Francis Fonyuy Tukov; Kyle Montgomery; Aaron Kleyn; Rui Sun; Sitra Tauscher-Wisniewski; Petra Kaufmann; Gerd Kullak-Ublick
Journal: J Hepatol Date: 2020-11-10 Impact factor: 25.083

5. Liver Injury Increases the Incidence of HCC following AAV Gene Therapy in Mice.

Authors: Dhwanil A Dalwadi; Laura Torrens; Jordi Abril-Fornaguera; Roser Pinyol; Catherine Willoughby; Jeffrey Posey; Josep M Llovet; Christian Lanciault; David W Russell; Markus Grompe; Willscott E Naugler
Journal: Mol Ther Date: 2020-10-22 Impact factor: 11.454

6. Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH.

Authors: Barbara Konkle; Glen Pierce; Donna Coffin; Mayss Naccache; R Cary Clark; Lindsey George; Alfonso Iorio; Brian O'Mahony; Steven Pipe; Mark Skinner; Crystal Watson; Flora Peyvandi; Johnny Mahlangu
Journal: J Thromb Haemost Date: 2020-11 Impact factor: 5.824

7. A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells.

Authors: Giang N Nguyen; John K Everett; Samita Kafle; Aoife M Roche; Hayley E Raymond; Jacob Leiby; Christian Wood; Charles-Antoine Assenmacher; Elizabeth P Merricks; C Tyler Long; Haig H Kazazian; Timothy C Nichols; Frederic D Bushman; Denise E Sabatino
Journal: Nat Biotechnol Date: 2020-11-16 Impact factor: 54.908

8. SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.

Authors: Christian Mueller; James D Berry; Diane M McKenna-Yasek; Gwladys Gernoux; Margaret A Owegi; Lindsay M Pothier; Catherine L Douthwright; Dario Gelevski; Sarah D Luppino; Meghan Blackwood; Nicholas S Wightman; Derek H Oakley; Matthew P Frosch; Terrence R Flotte; Merit E Cudkowicz; Robert H Brown
Journal: N Engl J Med Date: 2020-07-09 Impact factor: 91.245

Eliminating Panglossian thinking in development of AAV therapeutics.

1. Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1.

2. MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates.

3. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.

4. Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy.

5. Liver Injury Increases the Incidence of HCC following AAV Gene Therapy in Mice.

6. Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH.

7. A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells.

8. SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.

Review 9. Immune Responses to Viral Gene Therapy Vectors.

1. The Hemophilia Gene Therapy Patient Journey: Questions and Answers for Shared Decision-Making.

2. Treatment-induced hemophilic thrombosis?

3. Gene therapy - are we ready now?