Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a
Gene Therapy
by 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
3d ago
Gene Therapy, Published online: 25 March 2024; doi:10.1038/s41434-024-00449-x Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a ..read more
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Precision ophthalmology: a call for Africa not to be left in the dark
Gene Therapy
by Lisa Roberts
5d ago
Gene Therapy, Published online: 22 March 2024; doi:10.1038/s41434-024-00448-y Precision ophthalmology: a call for Africa not to be left in the dark ..read more
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Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Gene Therapy
by 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
1w ago
Gene Therapy, Published online: 14 March 2024; doi:10.1038/s41434-024-00446-0 Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation ..read more
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Analytical characterization of full, intermediate, and empty AAV capsids
Gene Therapy
by 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 Bruccoleri, Tim Kelly, James B. McGivney IV
1M ago
Gene Therapy, Published online: 19 February 2024; doi:10.1038/s41434-024-00444-2 Analytical characterization of full, intermediate, and empty AAV capsids ..read more
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Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia
Gene Therapy
by Ingrid Pabinger, Mila Ayash-Rashkovsky, Miguel Escobar, Barbara A. Konkle, María Eva Mingot-Castellano, Eric S. Mullins, Claude Negrier, Luying Pan, Kavitha Rajavel, Brian Yan, John Chapin
1M ago
Gene Therapy, Published online: 14 February 2024; doi:10.1038/s41434-024-00441-5 Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia ..read more
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Gene therapy corrects the neurological deficits of mice with sialidosis
Gene Therapy
by Wuh-Liang Hwu, Karine Chang, Yu-Han Liu, Hao-Chun Wang, Ni-Chung Lee, Yin-Hsiu Chien
1M ago
Gene Therapy, Published online: 07 February 2024; doi:10.1038/s41434-024-00443-3 Gene therapy corrects the neurological deficits of mice with sialidosis ..read more
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Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model RhoP23H+/−
Gene Therapy
by 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
2M ago
Gene Therapy, Published online: 26 January 2024; doi:10.1038/s41434-024-00440-6 Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model RhoP23H ..read more
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Development of a stable Sf9 insect cell line to produce VSV-G pseudotyped baculoviruses
Gene Therapy
by María del Pilar Plastine, Sabrina Amalfi, María Gabriela López, María José Gravisaco, Oscar Taboga, Victoria Alfonso
2M ago
Gene Therapy, Published online: 26 January 2024; doi:10.1038/s41434-024-00442-4 Development of a stable Sf9 insect cell line to produce VSV-G pseudotyped baculoviruses ..read more
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Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates
Gene Therapy
by 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, Thomas J. Cradick, Jennifer Gordon, Kamel Khalili
2M ago
Gene Therapy, Published online: 12 January 2024; doi:10.1038/s41434-023-00438-6 Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates ..read more
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AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes
Gene Therapy
by 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
2M ago
Gene Therapy, Published online: 10 January 2024; doi:10.1038/s41434-023-00436-8 AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes ..read more
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