Gene Therapy
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Gene Therapy covers both the research and clinical applications of novel therapeutic techniques based on a genetic component. Over the last few decades, significant advances in technologies ranging from identifying novel genetic targets that cause disease through to clinical studies, which show therapeutic benefit, have elevated this multidisciplinary field to the forefront of modern medicine.
Gene Therapy
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
Gene Therapy
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
Gene Therapy
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
Gene Therapy
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
Gene Therapy
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
Gene Therapy
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
Gene Therapy
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
Gene Therapy
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
Gene Therapy
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
Gene Therapy
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