Gene Therapy <em>Gene Therapy</em> 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. <p><em>Gene Therapy</em>&nbsp;covers all aspects of gene therapy as applied to human disease, including:</p> <ul> <li>preliminary studies which reveal novel genetic targets or strategies that improve a disease phenotype (Note: studies showing the mechanistic role of genetic elements alone, will be considered if significant therapeutic benefit or novelty is shown)</li> <li>the development of novel techniques for gene therapy research and improvements on established tools</li> <li>preclinical animal model systems and validation studies</li> <li>gene-based vaccine development and applications</li> <li>genetically engineered cellular models that can advance the validation of novel therapeutic targets, and drug discovery especially those using high-throughput applications and 3D modelling</li> <li>cell-based therapies including all aspects of stem cells and genetically modified cellular approaches (especially CRISPR and other gene editing strategies), including biobanking studies</li> <li>clinical trial reports which have significant impact for the field</li> <li>novel genetic biomarkers of disease susceptibility or disease progression with extensive scientific validation</li> <li>Industry sector pipeline profiles or commercial implications of gene therapies</li> <li>Regulatory landscapes and legislative issues pertaining to different global regions</li> </ul> http://feeds.nature.com/gt/rss/current Nature Publishing Group en © 2024 Macmillan Publishers Limited, part of Springer Nature. All rights reserved. Gene Therapy © 2024 Macmillan Publishers Limited, part of Springer Nature. All rights reserved. permissions@nature.com Gene Therapy https://www.nature.com/uploads/product/gt/rss.png http://feeds.nature.com/gt/rss/current <![CDATA[Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a]]> https://www.nature.com/articles/s41434-024-00449-x Gene Therapy, Published online: 25 March 2024; doi:10.1038/s41434-024-00449-x

Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a]]>
Zach HetzlerStella M. MarinakosNoah LottNoor MohammadAgnieszka Lass-NapiorkowskaJenna KolbeLauren TurrentineDelaney FieldsLaurie OvertonHelena MarieAngus HucknallOliver RammoHenry GeorgeQingshan Wei doi:10.1038/s41434-024-00449-x Gene Therapy, Published online: 2024-03-25; | doi:10.1038/s41434-024-00449-x 2024-03-25 Gene Therapy 10.1038/s41434-024-00449-x https://www.nature.com/articles/s41434-024-00449-x
<![CDATA[Precision ophthalmology: a call for Africa not to be left in the dark]]> https://www.nature.com/articles/s41434-024-00448-y 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]]>
Lisa Roberts doi:10.1038/s41434-024-00448-y Gene Therapy, Published online: 2024-03-22; | doi:10.1038/s41434-024-00448-y 2024-03-22 Gene Therapy 10.1038/s41434-024-00448-y https://www.nature.com/articles/s41434-024-00448-y
<![CDATA[Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation]]> https://www.nature.com/articles/s41434-024-00446-0 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]]>
Maria Elisabeth KallandTomas Pose-BoirazianGloria Maria PalomoFrauke Naumann-WinterEnrico CostaDarius MatuseviciusDinah M. DuarteEva MalikovaDinko VitezicKristina LarssonArmando MagrelliVioleta Stoyanova-BeninskaSegundo Mariz doi:10.1038/s41434-024-00446-0 Gene Therapy, Published online: 2024-03-14; | doi:10.1038/s41434-024-00446-0 2024-03-14 Gene Therapy 10.1038/s41434-024-00446-0 https://www.nature.com/articles/s41434-024-00446-0
<![CDATA[A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice]]> https://www.nature.com/articles/s41434-024-00447-z Gene Therapy, Published online: 12 March 2024; doi:10.1038/s41434-024-00447-z

A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice]]>
Felix KrauseKatja SchmidtkeMailton Franca de VasconcelosDavid SchmidtBeyza CansizFranziska TheisenMelanie D. MarkMax O. Rybarski doi:10.1038/s41434-024-00447-z Gene Therapy, Published online: 2024-03-12; | doi:10.1038/s41434-024-00447-z 2024-03-12 Gene Therapy 10.1038/s41434-024-00447-z https://www.nature.com/articles/s41434-024-00447-z
<![CDATA[Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate]]> https://www.nature.com/articles/s41434-024-00445-1 Gene Therapy, Published online: 01 March 2024; doi:10.1038/s41434-024-00445-1

Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate]]>
Mathieu GuerriaudCyril PoupetZineb LakhrifEvelyne KohliNathalie Moiré doi:10.1038/s41434-024-00445-1 Gene Therapy, Published online: 2024-03-01; | doi:10.1038/s41434-024-00445-1 2024-03-01 Gene Therapy 10.1038/s41434-024-00445-1 https://www.nature.com/articles/s41434-024-00445-1
<![CDATA[Analytical characterization of full, intermediate, and empty AAV capsids]]> https://www.nature.com/articles/s41434-024-00444-2 Gene Therapy, Published online: 19 February 2024; doi:10.1038/s41434-024-00444-2

Analytical characterization of full, intermediate, and empty AAV capsids]]>
Aisleen McColl-CarboniSerena DolliveSarah LaughlinRudenc LushiMichael MacArthurShanshan ZhouJeffrey GagnonChristopher A. SmithBrenda BurnhamRobert HortonDimpal LataBrianna UgaKalyani NatuEmmanuela MichelCelia SlaterEvan DaSilvaRobert BruccoleriTim KellyJames B. McGivney IV doi:10.1038/s41434-024-00444-2 Gene Therapy, Published online: 2024-02-19; | doi:10.1038/s41434-024-00444-2 2024-02-19 Gene Therapy 10.1038/s41434-024-00444-2 https://www.nature.com/articles/s41434-024-00444-2
<![CDATA[Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia]]> https://www.nature.com/articles/s41434-024-00441-5 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]]>
Ingrid PabingerMila Ayash-RashkovskyMiguel EscobarBarbara A. KonkleMaría Eva Mingot-CastellanoEric S. MullinsClaude NegrierLuying PanKavitha RajavelBrian YanJohn Chapin doi:10.1038/s41434-024-00441-5 Gene Therapy, Published online: 2024-02-14; | doi:10.1038/s41434-024-00441-5 2024-02-14 Gene Therapy 10.1038/s41434-024-00441-5 https://www.nature.com/articles/s41434-024-00441-5
<![CDATA[Gene therapy corrects the neurological deficits of mice with sialidosis]]> https://www.nature.com/articles/s41434-024-00443-3 Gene Therapy, Published online: 07 February 2024; doi:10.1038/s41434-024-00443-3

Gene therapy corrects the neurological deficits of mice with sialidosis]]>
Wuh-Liang HwuKarine ChangYu-Han LiuHao-Chun WangNi-Chung LeeYin-Hsiu Chien doi:10.1038/s41434-024-00443-3 Gene Therapy, Published online: 2024-02-07; | doi:10.1038/s41434-024-00443-3 2024-02-07 Gene Therapy 10.1038/s41434-024-00443-3 https://www.nature.com/articles/s41434-024-00443-3