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Select item 117855261.

Bridging gene therapy and next-generation vaccine technologies

Kristie Bloom, Abdullah Ely, Mohube Betty Maepa, Patrick Arbuthnot

Gene Ther. 2025; 32(1): 4–7. Published online 2024 Nov 18. doi: 10.1038/s41434-024-00502-9

PMCID:: PMC11785526

Article PDF–568K

Select item 115765082.

Gene therapy of Dent disease type 1 in newborn ClC-5 null mice for sustained transgene expression and gene therapy effects

Pin Lyu, Manish Kumar Yadav, Kyung Whan Yoo, Cuili Jiang, Qingqi Li, Anthony Atala, Baisong Lu

Gene Ther. 2024; 31(11-12): 563–571. Published online 2024 Sep 25. doi: 10.1038/s41434-024-00490-w

PMCID:: PMC11576508

Article PDF–2.6M

Select item 115765113.

BRD9 promotes the progression of gallbladder cancer via CST1 upregulation and interaction with FOXP1 through the PI3K/AKT pathway and represents a therapeutic target

Jing Qiang, Cheng Zhao, Liu-Qing Shi, Si-Rui Sun, Hua-Kai Wang, Shi-Lei Liu, Zi-Yi Yang, Ping Dong, Shan-Shan Xiang, Jian-Dong Wang, Yi-Jun Shu

Gene Ther. 2024; 31(11-12): 594–606. Published online 2024 Sep 21. doi: 10.1038/s41434-024-00488-4

PMCID:: PMC11576511

Article PDF–4.7M

Select item 115765044.

Correction: AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses

Henna Korpela, Jaakko Lampela, Jonna Airaksinen, Niko Järveläinen, Satu Siimes, Kaisa Valli, Tiina Nieminen, Minttu Turunen, Maria Grönman, Antti Saraste, Juhani Knuuti, Mikko Hakulinen, Pekka Poutiainen, Vesa Kärjä, Jussi Nurro, Seppo Ylä-Herttuala

Gene Ther. 2024; 31(11-12): 626–628. Published online 2024 Aug 26. doi: 10.1038/s41434-024-00481-x

Corrects:: Gene Ther. 2022; 29(10-11): 643–652.

PMCID:: PMC11576504

Correction PDF–1.0M

Select item 115765075.

Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models

Alexandra McCarron, Kak-Ming Ling, Samuel T. Montgomery, Kelly M. Martinovich, Patricia Cmielewski, Nathan Rout-Pitt, Anthony Kicic, David Parsons, Martin Donnelley

Gene Ther. 2024; 31(11-12): 553–559. Published online 2024 Aug 25. doi: 10.1038/s41434-024-00480-y

PMCID:: PMC11576507

Article PDF–1.7M

Select item 113991006.

Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model

Ivan Milenkovic, Shani Blumenreich, Ariel Hochfelder, Aviya Azulay, Inbal E. Biton, Mirie Zerbib, Roni Oren, Michael Tsoory, Tammar Joseph, Sarel J. Fleishman, Anthony H. Futerman

Gene Ther. 2024; 31(9-10): 439–444. Published online 2024 Aug 15. doi: 10.1038/s41434-024-00476-8

PMCID:: PMC11399100

Article PDF–1.5M

Select item 113990767.

Correction: Alternative oxidase encoded by sequence-optimized and chemically-modified RNA transfected into mammalian cells is catalytically active

Luca Giordano, Manish K. Aneja, Natascha Sommer, Nasim Alebrahimdehkordi, Alireza Seraji, Norbert Weissmann, Carsten Rudolph, Christian Plank, Howard T. Jacobs, Marten Szibor

Gene Ther. 2024; 31(9-10): 528. Published online 2024 Aug 15. doi: 10.1038/s41434-024-00473-x

Corrects:: Gene Ther. 2022; 29(12): 655–664.

PMCID:: PMC11399076

Correction PDF–279K

Select item 116001228.

The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2

Mengtian Cui, Qin Su, Mitchell Yip, Jackson McGowan, Claudio Punzo, Guangping Gao, Phillip W. L. Tai

Gene Ther. Author manuscript; available in PMC 2024 Nov 27.

Published in final edited form as: Gene Ther. 2024 Sep; 31(9-10): 489–498. Published online 2024 Aug 12. doi: 10.1038/s41434-024-00477-7

PMCID:: PMC11600122

Article PDF–1.6M

Select item 113990879.

Characterization of brain transduction capability of a BBB-penetrant AAV vector in mice, rats and macaques reveals differences in expression profiles

María Bunuales, Angeles Garduno, Miguel Chillon, Assumpció Bosch, Manuela Gonzalez-Aparicio, Maria Espelosin, Marta Garcia-Gomara, Alberto J. Rico, Ana Garcia-Osta, Mar Cuadrado-Tejedor, Jose L. Lanciego, Ruben Hernandez-Alcoceba

Gene Ther. 2024; 31(9-10): 455–466. Published online 2024 Jul 22. doi: 10.1038/s41434-024-00466-w

PMCID:: PMC11399087

Article PDF–8.6M

Select item 1125794510.

Retinoic acid related orphan receptor α is a genetic modifier that rescues retinal degeneration in a mouse model of Stargardt disease and Dry AMD

M. Akula, S. M. McNamee, Z. Love, N. Nasraty, N. P. M. Chan, M. Whalen, M. O. Avola, A. M. Olivares, B. D. Leehy, A. S. Jelcick, P. Singh, A. K. Upadhyay, D. F. Chen, N. B. Haider

Gene Ther. 2024; 31(7-8): 413–421. Published online 2024 May 16. doi: 10.1038/s41434-024-00455-z

PMCID:: PMC11257945

Article PDF–2.2M

Select item 1125794811.

Development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer into freshly isolated immune cells

Alexander Renner, Anika Stahringer, Katharina Eva Ruppel, Stephan Fricke, Ulrike Koehl, Dominik Schmiedel

Gene Ther. 2024; 31(7-8): 378–390. Published online 2024 Apr 29. doi: 10.1038/s41434-024-00454-0

PMCID:: PMC11257948

Article PDF–3.4M

Select item 1124595912.

Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease

Guo Ge, Barath P. Sivasubramanian, Bill D. Geng, Shujie Zhao, Qing Zhou, Gang Huang, Jason C. O’Connor, Robert A. Clark, Senlin Li

Gene Ther. Author manuscript; available in PMC 2024 Jul 13.

Published in final edited form as: Gene Ther. 2024 May; 31(5-6): 324–334. Published online 2024 Apr 16. doi: 10.1038/s41434-024-00451-3

PMCID:: PMC11245959

Article PDF–2.4M

Select item 1109078013.

Precision ophthalmology: a call for Africa not to be left in the dark

Lisa Roberts

Gene Ther. 2024; 31(5-6): 199–201. Published online 2024 Mar 22. doi: 10.1038/s41434-024-00448-y

PMCID:: PMC11090780

Article PDF–327K

Select item 1125796114.

Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation

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

Gene Ther. 2024; 31(7-8): 366–377. Published online 2024 Mar 14. doi: 10.1038/s41434-024-00446-0

PMCID:: PMC11257961

Article PDF–1013K

Select item 1109081015.

Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia

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

Gene Ther. 2024; 31(5-6): 273–284. Published online 2024 Feb 14. doi: 10.1038/s41434-024-00441-5

PMCID:: PMC11090810

Article PDF–918K

Select item 1109081516.

Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model Rho^P23H+/⁻

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

Gene Ther. 2024; 31(5-6): 255–262. Published online 2024 Jan 26. doi: 10.1038/s41434-024-00440-6

PMCID:: PMC11090815

Article PDF–1.9M

Select item 1125795117.

Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates

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

Gene Ther. 2024; 31(7-8): 434–435. Published online 2024 Jan 12. doi: 10.1038/s41434-023-00438-6