Gene therapy

Keyphrases

Publications

volume 31, number 5-6, 2024

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
Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model Rho P23H+/ . Gene therapy 31 (5-6) (2024)
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
Analytical characterization of full, intermediate, and empty AAV capsids. Gene therapy 31 (5-6) (2024)
Hyeokgyo Jeong, Sujata Purja, Eun Young Kim
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 31 (5-6) (2024)
Ingrid Pabinger, Mila Ayash-Rashkovsky, Miguel A Escobar, Barbara A Konkle, Maria-Eva Mingot-Castellano, Eric S Mullins, Claude G Negrier, Luying Pan, Kavitha Rajavel, Brian Yan, John C Chapin
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia. Gene therapy 31 (5-6) (2024)
Felix Krause, Katja Schmidtke, Mailton Franca de Vasconcelos, David Schmidt, Beyza Cansiz, Franziska Theisen, Melanie D Mark, Max O Rybarski
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice. Gene therapy 31 (5-6) (2024)
Paul Wuh-Liang Hwu, Karine Chang, Yu-Han Liu, Hao-Chun Wang, Ni-Chung Lee, Yin-Hsiu Chien
Gene therapy corrects the neurological deficits of mice with sialidosis. Gene therapy 31 (5-6) (2024)
Mathieu Guerriaud, Cyril Poupet, Zineb Lakhrif, Evelyne Kohli, Nathalie Moiré
Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate. Gene therapy 31 (5-6) (2024)
Paul Wuh-Liang Hwu, Karine Chang, Yu-Han Liu, Hao-Chun Wang, Ni-Chung Lee, Yin-Hsiu Chien
Gene therapy corrects the neurological deficits of mice with sialidosis. Gene therapy 31 (5-6) (2024)
Ingrid Pabinger, Mila Ayash-Rashkovsky, Miguel A Escobar, Barbara A Konkle, Maria-Eva Mingot-Castellano, Eric S Mullins, Claude G Negrier, Luying Pan, Kavitha Rajavel, Brian Yan, John C Chapin
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia. Gene therapy 31 (5-6) (2024)
Shahin Eghbalsaied, Clancy Lawler, Björn Petersen, Raul A Hajiyev, Steven R Bischoff, Stephen R Frankenberg
CRISPR/Cas9-mediated base editors and their prospects for mitochondrial genome engineering. Gene therapy 31 (5-6) (2024)
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
Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a. Gene therapy 31 (5-6) (2024)
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
Analytical characterization of full, intermediate, and empty AAV capsids. Gene therapy 31 (5-6) (2024)
Mathieu Guerriaud, Cyril Poupet, Zineb Lakhrif, Evelyne Kohli, Nathalie Moiré
Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate. Gene therapy 31 (5-6) (2024)
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
Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model Rho P23H+/ . Gene therapy 31 (5-6) (2024)
Alexis Ceecee Zhang, Myra B McGuiness, Fred K Chen, John R B Grigg, Heather G Mack, Lauren N Ayton
A multinational survey of potential participant perspectives on ocular gene therapy. Gene therapy 31 (5-6) (2024)
Lisa Roberts
Precision ophthalmology: a call for Africa not to be left in the dark. Gene therapy 31 (5-6) (2024)
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
Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a. Gene therapy 31 (5-6) (2024)
Hyeokgyo Jeong, Sujata Purja, Eun Young Kim
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 31 (5-6) (2024)
Lisa Roberts
Precision ophthalmology: a call for Africa not to be left in the dark. Gene therapy 31 (5-6) (2024)
Felix Krause, Katja Schmidtke, Mailton Franca de Vasconcelos, David Schmidt, Beyza Cansiz, Franziska Theisen, Melanie D Mark, Max O Rybarski
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice. Gene therapy 31 (5-6) (2024)
Shahin Eghbalsaied, Clancy Lawler, Björn Petersen, Raul A Hajiyev, Steven R Bischoff, Stephen R Frankenberg
CRISPR/Cas9-mediated base editors and their prospects for mitochondrial genome engineering. Gene therapy 31 (5-6) (2024)
Lisa Roberts
Precision ophthalmology: a call for Africa not to be left in the dark. Gene therapy 31 (5-6) (2024)
Guo Ge, Barath P Sivasubramanian, Bill D Geng, Shujie Zhao, Qing Zhou, Gang Huang, Jason C O'Connor, Robert A Clark, Senlin Li
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 31 (5-6) (2024)
Guo Ge, Barath P Sivasubramanian, Bill D Geng, Shujie Zhao, Qing Zhou, Gang Huang, Jason C O'Connor, Robert A Clark, Senlin Li
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 31 (5-6) (2024)
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
Analytical characterization of full, intermediate, and empty AAV capsids. Gene therapy 31 (5-6) (2024)
Felix Krause, Katja Schmidtke, Mailton Franca de Vasconcelos, David Schmidt, Beyza Cansiz, Franziska Theisen, Melanie D Mark, Max O Rybarski
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice. Gene therapy 31 (5-6) (2024)
Ingrid Pabinger, Mila Ayash-Rashkovsky, Miguel A Escobar, Barbara A Konkle, Maria-Eva Mingot-Castellano, Eric S Mullins, Claude G Negrier, Luying Pan, Kavitha Rajavel, Brian Yan, John C Chapin
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia. Gene therapy 31 (5-6) (2024)
Guo Ge, Barath P Sivasubramanian, Bill D Geng, Shujie Zhao, Qing Zhou, Gang Huang, Jason C O'Connor, Robert A Clark, Senlin Li
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 31 (5-6) (2024)
Alexis Ceecee Zhang, Myra B McGuiness, Fred K Chen, John R B Grigg, Heather G Mack, Lauren N Ayton
A multinational survey of potential participant perspectives on ocular gene therapy. Gene therapy 31 (5-6) (2024)
Alexis Ceecee Zhang, Myra B McGuiness, Fred K Chen, John R B Grigg, Heather G Mack, Lauren N Ayton
A multinational survey of potential participant perspectives on ocular gene therapy. Gene therapy 31 (5-6) (2024)
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
Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model Rho P23H+/ . Gene therapy 31 (5-6) (2024)

volume 31, number 7-8, 2024

Monica 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, Dong Feng Chen, Neena B Haider
Retinoic acid related orphan receptor α is a genetic modifier that rescues retinal degeneration in a mouse model of Stargardt disease and Dry AMD. Gene therapy 31 (7-8) (2024)
Maria Elisabeth Kalland, Tomas Pose-Boirazian, Gloria Maria Palomo, Frauke Naumann-Winter, Enrico Costa, Darius Matusevicius, Dinah M Duarte, Eva Malikova, Dinko Vitezić, Kristina Larsson, Armando Magrelli, Violeta Stoyanova-Beninska, Segundo Mariz
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation. Gene therapy 31 (7-8) (2024)
Alexander Renner, Anika Stahringer, Katharina Eva Ruppel, Stephan Fricke, Ulrike Köhl, Dominik Schmiedel
Development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer into freshly isolated immune cells. Gene therapy 31 (7-8) (2024)
Rodrigo de Holanda Mendonça, Adriana Banzzatto Ortega, Ciro Matsui, Vanessa van der Linden, Marcelo Kerstenetzky, Luis Fernando Grossklauss, Elizabeth L Silveira-Lucas, Graziela Jorge Polido, Edmar Zanoteli
Gene replacement therapy for spinal muscular atrophy: safety and preliminary efficacy in a Brazilian cohort. Gene therapy 31 (7-8) (2024)
Alexander Renner, Anika Stahringer, Katharina Eva Ruppel, Stephan Fricke, Ulrike Köhl, Dominik Schmiedel
Development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer into freshly isolated immune cells. Gene therapy 31 (7-8) (2024)
Monica 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, Dong Feng Chen, Neena B Haider
Retinoic acid related orphan receptor α is a genetic modifier that rescues retinal degeneration in a mouse model of Stargardt disease and Dry AMD. Gene therapy 31 (7-8) (2024)
Jordyn A Lopes, Nicole E Garnier, Yanlong Pei, Jacob G E Yates, Elena S B Campbell, Melanie M Goens, Madison E Hughes, Amira D Rghei, Brenna A Y Stevens, Matthew M Guilleman, Brad Thompson, Cezar M Khursigara, Leonardo Susta, Sarah K Wootton
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia. Gene therapy 31 (7-8) (2024)
Maria Elisabeth Kalland, Tomas Pose-Boirazian, Gloria Maria Palomo, Frauke Naumann-Winter, Enrico Costa, Darius Matusevicius, Dinah M Duarte, Eva Malikova, Dinko Vitezić, Kristina Larsson, Armando Magrelli, Violeta Stoyanova-Beninska, Segundo Mariz
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation. Gene therapy 31 (7-8) (2024)
Zhuang Mao, Changyao Wang, Juanli Liu, Xue Li, Han Duan, Yi Ye, Huifang Liu, Lin Lv, Guanzhen Xue, Zhichao He, Ta-Na Wuren, Hua Wang
Superoxide dismutase 1-modified dental pulp stem cells alleviate high-altitude pulmonary edema by inhibiting oxidative stress through the Nrf2/HO-1 pathway. Gene therapy 31 (7-8) (2024)
Monica 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, Dong Feng Chen, Neena B Haider
Retinoic acid related orphan receptor α is a genetic modifier that rescues retinal degeneration in a mouse model of Stargardt disease and Dry AMD. Gene therapy 31 (7-8) (2024)
Rodrigo de Holanda Mendonça, Adriana Banzzatto Ortega, Ciro Matsui, Vanessa van der Linden, Marcelo Kerstenetzky, Luis Fernando Grossklauss, Elizabeth L Silveira-Lucas, Graziela Jorge Polido, Edmar Zanoteli
Gene replacement therapy for spinal muscular atrophy: safety and preliminary efficacy in a Brazilian cohort. Gene therapy 31 (7-8) (2024)
Alexander Renner, Anika Stahringer, Katharina Eva Ruppel, Stephan Fricke, Ulrike Köhl, Dominik Schmiedel
Development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer into freshly isolated immune cells. Gene therapy 31 (7-8) (2024)
Maria Elisabeth Kalland, Tomas Pose-Boirazian, Gloria Maria Palomo, Frauke Naumann-Winter, Enrico Costa, Darius Matusevicius, Dinah M Duarte, Eva Malikova, Dinko Vitezić, Kristina Larsson, Armando Magrelli, Violeta Stoyanova-Beninska, Segundo Mariz
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation. Gene therapy 31 (7-8) (2024)
Zhuang Mao, Changyao Wang, Juanli Liu, Xue Li, Han Duan, Yi Ye, Huifang Liu, Lin Lv, Guanzhen Xue, Zhichao He, Ta-Na Wuren, Hua Wang
Superoxide dismutase 1-modified dental pulp stem cells alleviate high-altitude pulmonary edema by inhibiting oxidative stress through the Nrf2/HO-1 pathway. Gene therapy 31 (7-8) (2024)
Monica 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, Dong Feng Chen, Neena B Haider
Retinoic acid related orphan receptor α is a genetic modifier that rescues retinal degeneration in a mouse model of Stargardt disease and Dry AMD. Gene therapy 31 (7-8) (2024)
Johanna Risse, Merlin Krzemien, Jan Schnalke, Thomas Heinemann
Towards ethical drug pricing: the European Orphan Genomic Therapies Fund. Gene therapy 31 (7-8) (2024)
Johanna Risse, Merlin Krzemien, Jan Schnalke, Thomas Heinemann
Towards ethical drug pricing: the European Orphan Genomic Therapies Fund. Gene therapy 31 (7-8) (2024)
Johanna Risse, Merlin Krzemien, Jan Schnalke, Thomas Heinemann
Towards ethical drug pricing: the European Orphan Genomic Therapies Fund. Gene therapy 31 (7-8) (2024)
Yeounsun Oh, Lee Wha Gwon, Hyomin K Lee, Junho K Hur, Kwang-Hyun Park, Kee-Pyo Kim, Seung Hwan Lee
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system. Gene therapy 31 (7-8) (2024)