Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease
Gene Therapy, Published online: 16 April 2024; doi:10.1038/s41434-024-00451-3Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease (Source: Gene Therapy)
Source: Gene Therapy - April 16, 2024 Category: Genetics & Stem Cells Authors: Guo Ge Barath P. Sivasubramanian Bill D. Geng Shujie Zhao Qing Zhou Gang Huang Jason C. O ’Connor Robert A. Clark Senlin Li Source Type: research
A multinational survey of potential participant perspectives on ocular gene therapy
Gene Therapy, Published online: 02 April 2024; doi:10.1038/s41434-024-00450-4A multinational survey of potential participant perspectives on ocular gene therapy (Source: Gene Therapy)
Source: Gene Therapy - April 2, 2024 Category: Genetics & Stem Cells Authors: Alexis Ceecee Britten-Jones Myra B. McGuinness Fred K. Chen John R. Grigg Heather G. Mack Lauren N. Ayton Source Type: research
Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a
Gene Therapy, Published online: 25 March 2024; doi:10.1038/s41434-024-00449-xAdeno-associated virus genome quantification with amplification-free CRISPR-Cas12a (Source: Gene Therapy)
Source: Gene Therapy - March 25, 2024 Category: Genetics & Stem Cells Authors: Zach Hetzler Stella M. Marinakos Noah Lott Noor Mohammad Agnieszka Lass-Napiorkowska Jenna Kolbe Lauren Turrentine Delaney Fields Laurie Overton Helena Marie Angus Hucknall Oliver Rammo Henry George Qingshan Wei Source Type: research
Precision ophthalmology: a call for Africa not to be left in the dark
Gene Therapy, Published online: 22 March 2024; doi:10.1038/s41434-024-00448-yPrecision ophthalmology: a call for Africa not to be left in the dark (Source: Gene Therapy)
Source: Gene Therapy - March 22, 2024 Category: Genetics & Stem Cells Authors: Lisa Roberts Source Type: research
Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Gene Therapy, Published online: 14 March 2024; doi:10.1038/s41434-024-00446-0Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation (Source: Gene Therapy)
Source: Gene Therapy - March 14, 2024 Category: Genetics & Stem Cells Authors: Maria Elisabeth Kalland Tomas Pose-Boirazian Gloria Maria Palomo Frauke Naumann-Winter Enrico Costa Darius Matusevicius Dinah M. Duarte Eva Malikova Dinko Vitezic Kristina Larsson Armando Magrelli Violeta Stoyanova-Beninska Segundo Mariz Source Type: research
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice
Gene Therapy, Published online: 12 March 2024; doi:10.1038/s41434-024-00447-zA shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice (Source: Gene Therapy)
Source: Gene Therapy - March 12, 2024 Category: Genetics & Stem Cells Authors: Felix Krause Katja Schmidtke Mailton Franca de Vasconcelos David Schmidt Beyza Cansiz Franziska Theisen Melanie D. Mark Max O. Rybarski Source Type: research
Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate
Gene Therapy, Published online: 01 March 2024; doi:10.1038/s41434-024-00445-1Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate (Source: Gene Therapy)
Source: Gene Therapy - March 1, 2024 Category: Genetics & Stem Cells Authors: Mathieu Guerriaud Cyril Poupet Zineb Lakhrif Evelyne Kohli Nathalie Moir é Source Type: research
Analytical characterization of full, intermediate, and empty AAV capsids
Gene Therapy, Published online: 19 February 2024; doi:10.1038/s41434-024-00444-2Analytical characterization of full, intermediate, and empty AAV capsids (Source: Gene Therapy)
Source: Gene Therapy - February 19, 2024 Category: Genetics & Stem Cells Authors: Aisleen McColl-Carboni Serena Dollive Sarah Laughlin Rudenc Lushi Michael MacArthur Shanshan Zhou Jeffrey Gagnon Christopher A. Smith Brenda Burnham Robert Horton Dimpal Lata Brianna Uga Kalyani Natu Emmanuela Michel Celia Slater Evan DaSilva Robert Brucc Source Type: research
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia
Gene Therapy, Published online: 14 February 2024; doi:10.1038/s41434-024-00441-5Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia (Source: Gene Therapy)
Source: Gene Therapy - February 14, 2024 Category: Genetics & Stem Cells Authors: Ingrid Pabinger Mila Ayash-Rashkovsky Miguel Escobar Barbara A. Konkle Mar ía Eva Mingot-Castellano Eric S. Mullins Claude Negrier Luying Pan Kavitha Rajavel Brian Yan John Chapin Source Type: research
Gene therapy corrects the neurological deficits of mice with sialidosis
Gene Therapy, Published online: 07 February 2024; doi:10.1038/s41434-024-00443-3Gene therapy corrects the neurological deficits of mice with sialidosis (Source: Gene Therapy)
Source: Gene Therapy - February 7, 2024 Category: Genetics & Stem Cells Authors: Wuh-Liang Hwu Karine Chang Yu-Han Liu Hao-Chun Wang Ni-Chung Lee Yin-Hsiu Chien Source Type: research
Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model RhoP23H+/−
Gene Therapy, Published online: 26 January 2024; doi:10.1038/s41434-024-00440-6Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model RhoP23H+/− (Source: Gene Therapy)
Source: Gene Therapy - January 26, 2024 Category: Genetics & Stem Cells Authors: Shannon M. McNamee Natalie P. Chan Monica Akula Marielle O. Avola Maiya Whalen Kaden Nystuen Pushpendra Singh Arun K. Upadhyay Margaret M. DeAngelis Neena B. Haider Source Type: research
Development of a stable Sf9 insect cell line to produce VSV-G pseudotyped baculoviruses
Gene Therapy, Published online: 26 January 2024; doi:10.1038/s41434-024-00442-4Development of a stable Sf9 insect cell line to produce VSV-G pseudotyped baculoviruses (Source: Gene Therapy)
Source: Gene Therapy - January 26, 2024 Category: Genetics & Stem Cells Authors: Mar ía del Pilar Plastine Sabrina Amalfi Mar ía Gabriela López Mar ía José Gravisaco Oscar Taboga Victoria Alfonso Source Type: research
Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates
Gene Therapy, Published online: 12 January 2024; doi:10.1038/s41434-023-00438-6Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates (Source: Gene Therapy)
Source: Gene Therapy - January 12, 2024 Category: Genetics & Stem Cells Authors: Tricia H. Burdo Chen Chen Rafal Kaminski Ilker K. Sariyer Pietro Mancuso Martina Donadoni Mandy D. Smith Rahsan Sariyer Maurizio Caocci Shuren Liao Hong Liu Wenwen Huo Huaqing Zhao John Misamore Mark G. Lewis Vahan Simonyan Elaine E. Thompson Ethan Y. Xu Source Type: research
AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes
Gene Therapy, Published online: 10 January 2024; doi:10.1038/s41434-023-00436-8AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes (Source: Gene Therapy)
Source: Gene Therapy - January 10, 2024 Category: Genetics & Stem Cells Authors: Brahim Chaqour Thu T. Duong Jipeng Yue Tehui Liu David Camacho Kimberly E. Dine Julian Esteve-Rudd Scott Ellis Jean Bennett Kenneth S. Shindler Ahmara G. Ross Source Type: research
Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea
Gene Therapy, Published online: 10 January 2024; doi:10.1038/s41434-023-00437-7Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea (Source: Gene Therapy)
Source: Gene Therapy - January 10, 2024 Category: Genetics & Stem Cells Authors: Hyeokgyo Jeong Sujata Purja Eunyoung Kim Source Type: research
