logo
CanyamAI summaries for academic research
language
MobileMobile
  • Phone FeatureWeChat Mini Program
EnglishEnglish
noticeNotice
BlogBlog
delete
arrow
BackJournal Details
M

Molecular Therapy Methods and Clinical Development

IF4.7
Papers247
Citations6169
Journal Papers 229
Publication Date
delete
Genome Editing in Patient iPSCs Corrects the Most Prevalent USH2A Mutations and Reveals Intriguing Mutant mRNA Expression Profiles
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-24
delete0
deleteOAAI
deleteCarla Sanjurjo-Soriano; Nejla Erkilic; David Baux; Daria Mamaeva; Christian P. Hamel; Isabelle Meunier; Anne-Françoise Roux; Vasiliki Kalatzis
deleteShare
deleteSave
HBVZ10, An AAV8 Vector-Based New HBV Therapy Candidate for cccDNA Elimination
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-19
delete0
deleteOAAI
deleteBai-Hua Zhang; Yuanping Zhou; Stephen Horrigan; Laura Luckenbaugh; Jianming Hu; Fabien Zoulim; Yong-Yuan Zhang
deleteShare
deleteSave
Molecular Therapy Advances: Building the bridge between discovery and cure
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-17
delete0
deleteOAAI
deleteMohamed Abou-el-Enein
deleteShare
deleteSave
Unveiling Genomic Rearrangements in Engineered iPSC Lines by Optical Genome Mapping
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-15
delete0
deleteOAAI
deleteDarren Finlay; Pooja Hor; Benjamin H. Goldenson; Xiao-Hua Li; Rabi Murad; Dan S. Kaufman; Kristiina Vuori
deleteShare
deleteSave
Inhibition of immune response reduces pathology in dorsal root ganglia and peripheral nerves in cynomolgus macaques following adeno-associated virus gene therapy
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-14
delete0
deleteOAAI
deleteBranka Grubor; Kate L. Henry; Su Jing Chan; Mark Sheehan; Anumeha Shah; Alex Pellerin; Judy Bai; Prasad Nadella; Santhosh Bommegowda; Patrick Cullen; Eric Tien; Vivian Chen; Nicholas P. van der Munnik; Stephanie White-Hunt; Edward D. Plowey; Stefan Hamann; Amanda J. Guise; Shanqin Xu; Melissa Kirkland; Jessica Doherty
deleteShare
deleteSave
Modified AAV5 Capsid for Improved Brain Biodistribution Following Direct Injection in Preclinical Models
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-14
delete0
deleteOAAI
deleteSebastian N. Kieper; Elisabeth A. Spronck; Meriem Bourajjaj; Roberto D.V.S Morais; M. Leontien van der Bent; Seyda Acar Broekmans; Charlot van Rooijen; Rhodé van Westen; Giorgia Squeri; Giso Brasser; Morgane Wartel; Stijn J.H. Waaijer; C.Warner Hoornenborg; Tycho M. Hoogland; Richard Porter; Angga kusuma
deleteShare
deleteSave
Leveraging the Active Conformation of LFA-1 as a Potential Target for Hematological Malignancies
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-12
delete0
deleteOAAI
deleteAbhishek Puthenveetil; Guy Biber; Batel Sabag; Meirav Kedmi; Avigdor Abraham; Noah Joseph; Mira Barda-Saad
deleteShare
deleteSave
AAV-PHP.eB achieves superior neuronal transduction compared to AAV9 in pigtail macaques following intracerebroventricular administration.
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-12
delete0
deleteOAAI
deleteMichal G. Fortuna; Lyle H. Nyberg; Naz Taskin; Avery Hunker; Natalie Weed; Kathryn Gudsnuk; Melissa Berg; Boaz Levi; Ed Lein; Jonathan T. Ting
deleteShare
deleteSave
CRISPR/Methotrexate-integrated Strategy for TCR-T Cell Engineering with Reduced Chromosome 14 Loss
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-12
delete0
deleteOAAI
deleteJian Xu; Lianghua Shen; Ziyu Chen; Changmeng Zhang; Xiaodan Ding; Qiaomei He; Xiao Zhou; Peiyao Jiang; Jiayu Liu; Lu Li; Jinan Fang; Fanlin Li; Liping Wan; Xueying Ding; Yuqin Yang; Xiaorui Wang; Pengran Wang; Xianmin Song; Yan Zhang
deleteShare
deleteSave
Elucidation of the Binding Interaction Interface Between Adeno-Associated Virus Serotype 11 Capsid Protein and Host Nuclear Import Proteins
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-10
delete0
deleteOAAI
deleteMikayla Hoad; Sepehr Nematollahzadeh; Justin A. Roby; Gualtiero Alvisi; Jade K. Forwood
deleteShare
deleteSave
The Amazing AAV Capsids: Into the Structure-Verse
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-10
delete0
deleteOAAI
deleteJane His-Bell; Mario Mietzsch; Robert McKenna
deleteShare
deleteSave
Nonclinical Toxicity Study Duration in AAV Gene Therapy Development: Evidence from Industry Survey Supports Adequacy of Short-Term Assessments
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-10
delete0
deleteOAAI
deleteBasel T. Assaf; David Edwards; Shawn Jay Berens; Brian Lauritzen; Christelle Pierrot; Francis Fonyuy Tukov; Cristina Bertinetti-Lapatki; Manuela Braun; Kai Schaefer; Jessica L. Lynch
deleteShare
deleteSave
Retinal organoids mirror CRISPR/Cas9 gene editing efficiency observed in vivo
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-10
delete0
deleteOAAI
deleteJuliette Pulman; Hugo Malki; Paul Oudin; Ecem Aydin; Sophie Tran; Laura Visticot; Camille Robert; Anne De Cian; Marie As; Olivier Goureau; Jean-Paul Concordet; Deniz Dalkara
deleteShare
deleteSave
Targeted Inhibition of ELANE Expression Using Adenine Base Editing to Treat Severe Congenital Neutropenia
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-05
delete0
deleteOAAI
deleteBetül Findik; Benjamin Dannenmann; Franka Bernhard; Masako Monika Kaufmann; Sandra Ammann; Sergey Kandabarau; Maksim Klimiankou; Fabian Mauch; Patrick Münzer; Oliver Borst; Isabel Klefenz; Doris Steinemann; Claudia Lengerke; Cornelia Zeidler; Toni Cathomen; Karl Welte; Masoud Nasri; Julia Skokowa
deleteShare
deleteSave
Safety and Efficacy of AAV8-aflibercept in Treating Choroidal Neovascularization via Single-Cell RNA Sequencing
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-05
delete0
deleteOAAI
deleteJiemei Shi; Kaicheng Wu; Lihui Chen; Yinling Li; Chunhui Jiang
deleteShare
deleteSave
End-to-end characterization of AAV manufacturing process using charge detection mass spectrometry
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-11-05
delete0
deleteOAAI
deleteLohra M. Young; Benjamin E. Draper; Martin F. Jarrold; Clifford A. Froelich; Seyed Pouria Motevalian
deleteShare
deleteSave
Characterization of Difficult-to-Remove Host Cell Proteins in Adeno-Associated Virus Downstream Processing
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-10-30
delete0
deleteOAAI
deleteThomas M. Leibiger; Lie Min; Kelvin H. Lee
deleteShare
deleteSave
Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-10-30
delete0
deleteOAAI
deleteChiara Rigamonti; Dimitri Bulté; Federica Barzaghi; Cristina Mesa-Nuñez; Luca Basso-Ricci; Emanuela Pettinato; Camilla Visconti; Sara Degl’Innocenti; Francesco Gazzo; Fabrizio Benedicenti; Sergio Arévalo; Pamela Quaranta; Francesca Sanvito; Roberta Caorsi; Francesca Schena; Michela Lupia; Silvia Federici; Antonella Insalaco; Fabrizio De Benedetti; Serena Scala; Maurizio Miano; Francesca Conti; Maria Pia Cicalese; Eugenio Montini; Marco Gattorno; Carlo Dufour; Alessandro Aiuti; Alessandra Mortellaro
deleteShare
deleteSave
Intrathalamic delivery of adeno-associated viral vector expressing progranulin as gene therapy for GRN-related frontotemporal dementia
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-10-30
delete0
deleteOAAI
deleteYoun Bok Lee; Do Young Lee; Zoe Walker; Joseph Allison; Aleksandra Kaliszewska; Alinda Fernandes; Katharine Y. Zhang; Yalem Bekele; Paniz Hosseini; Romain Joubert; Joana Furtado; McQuinn Gumus; Olivier Brock; Ali Bienemann; Steven Gill; Samantha Murray; Nadia Mitchell; Page Bouchard; Alex Bloom; John Isaac; Carlos J. Miranda; Christopher E. Shaw
deleteShare
deleteSave
Nanobody-based gene therapy targeting complement component C3 reduces choroidal neovascularization in mice
deleteMolecular Therapy Methods & Clinical Development
IF4.7
delete2025-10-25
delete0
deleteOAAI
deleteEmilie Grarup Jensen; Thomas Stax Jakobsen; Gloriane Schnabolk; Kyrie Wilson; Mathias Rask-Pedersen; Nanna Jensen; Gregers Rom Andersen; Steffen Thiel; Lars Aagaard; Bärbel Rohrer; Anne Louise Askou; Thomas J. Corydon
deleteShare
deleteSave