Literature DB >> 34599291

Gene therapy community grapples with toxicity issues, as pipeline matures.

Asher Mullard.   

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Year:  2021        PMID： 34599291     DOI： 10.1038/d41573-021-00164-x

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


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  11 in total

1.  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 2.  Genetic pain loss disorders.

Authors:  Annette Lischka; Petra Lassuthova; Arman Çakar; Christopher J Record; Jonas Van Lent; Jonathan Baets; Maike F Dohrn; Jan Senderek; Angelika Lampert; David L Bennett; John N Wood; Vincent Timmerman; Thorsten Hornemann; Michaela Auer-Grumbach; Yesim Parman; Christian A Hübner; Miriam Elbracht; Katja Eggermann; C Geoffrey Woods; James J Cox; Mary M Reilly; Ingo Kurth
Journal:  Nat Rev Dis Primers       Date:  2022-06-16       Impact factor: 65.038

3.  Changing trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapies.

Authors:  Tamara Burdett; Samir Nuseibeh
Journal:  Gene Ther       Date:  2022-09-12       Impact factor: 4.184

4.  Serum extracellular vesicles for delivery of CRISPR-CAS9 ribonucleoproteins to modify the dystrophin gene.

Authors:  Nathalie Majeau; Annabelle Fortin-Archambault; Catherine Gérard; Joël Rousseau; Pouiré Yaméogo; Jacques P Tremblay
Journal:  Mol Ther       Date:  2022-05-26       Impact factor: 12.910

5.  AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities.

Authors:  J Fraser Wright
Journal:  Mol Ther       Date:  2022-05-20       Impact factor: 12.910

Review 6.  Immunogenicity and toxicity of AAV gene therapy.

Authors:  Hildegund C J Ertl
Journal:  Front Immunol       Date:  2022-08-12       Impact factor: 8.786

7.  Genome editing-mediated knock-in of therapeutic genes ameliorates the disease phenotype in a model of hemophilia.

Authors:  Jeong Hyeon Lee; Hye-Kyung Oh; Beom Seok Choi; Ho Hyeon Lee; Kyu Jun Lee; Un Gi Kim; Jina Lee; Hyerim Lee; Geon Seong Lee; Se Jun Ahn; Jeong Pil Han; Seokjoong Kim; Su Cheong Yeom; Dong Woo Song
Journal:  Mol Ther Nucleic Acids       Date:  2022-08-04       Impact factor: 10.183

Review 8.  Drug development progress in duchenne muscular dystrophy.

Authors:  Jiexin Deng; Junshi Zhang; Keli Shi; Zhigang Liu
Journal:  Front Pharmacol       Date:  2022-07-22       Impact factor: 5.988

9.  Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing.

Authors:  Katherine E Simon; Leo O Blondel; Trevor J Gonzalez; Marco M Fanous; Angela L Roger; Maribel Santiago Maysonet; Garth W Devlin; Timothy J Smith; Daniel K Oh; L Patrick Havlik; Ruth M Castellanos Rivera; Jorge A Piedrahita; Mai K ElMallah; Charles A Gersbach; Aravind Asokan
Journal:  Nat Commun       Date:  2022-10-10       Impact factor: 17.694

10.  Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells.

Authors:  Samuele Ferrari; Aurelien Jacob; Daniela Cesana; Marianne Laugel; Stefano Beretta; Angelica Varesi; Giulia Unali; Anastasia Conti; Daniele Canarutto; Luisa Albano; Andrea Calabria; Valentina Vavassori; Carlo Cipriani; Maria Carmina Castiello; Simona Esposito; Chiara Brombin; Federica Cugnata; Oumeya Adjali; Eduard Ayuso; Ivan Merelli; Anna Villa; Raffaella Di Micco; Anna Kajaste-Rudnitski; Eugenio Montini; Magalie Penaud-Budloo; Luigi Naldini
Journal:  Cell Stem Cell       Date:  2022-10-06       Impact factor: 25.269
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