Molecular therapy. Methods & clinical development

Keyphrases

Publications

volume 32, number 3, 2024

Hikari Okada, Takeharu Sakamoto, Kouki Nio, Yingyi Li, Kazuyuki Kuroki, Saiho Sugimoto, Tetsuro Shimakami, Nobuhide Doi, Masao Honda, Motoharu Seiki, Shuichi Kaneko, Taro Yamashita
Lipid nanoparticle-encapsulated DOCK11-siRNA efficiently reduces hepatitis B virus cccDNA level in infected mice. Molecular therapy. Methods & clinical development 32 (3) (2024)
Yoshiki Nakashima, Masayoshi Tsukahara
Atelocollagen supports three-dimensional culture of human induced pluripotent stem cells. Molecular therapy. Methods & clinical development 32 (3) (2024)
Ashrafali Mohamed Ismail, Evan Witt, Taren Bouwman, Wyatt Clark, Bridget Yates, Matteo Franco, Sylvia Fong
The longitudinal kinetics of AAV5 vector integration profiles and evaluation of clonal expansion in mice. Molecular therapy. Methods & clinical development 32 (3) (2024)
Ramya Rallabandi, Brenna Sharp, Spencer Majerus, Austin Royster, Sarrianna Hoffer, Mia Ikeda, Patricia Devaux
Engineering single-cycle MeV vector for CRISPR-Cas9 gene editing. Molecular therapy. Methods & clinical development 32 (3) (2024)
Robert D E Clark, Felix Rabito, Ferris T Munyonho, T Parks Remcho, Jay K Kolls
Evaluation of anti-vector immune responses to adenovirus-mediated lung gene therapy and modulation by αCD20. Molecular therapy. Methods & clinical development 32 (3) (2024)
Shin Jeon, Jaein Park, Shibi Likhite, Ji Hwan Moon, Dongjun Shin, Liwen Li, Kathrin C Meyer, Jae W Lee, Soo-Kyung Lee
The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome. Molecular therapy. Methods & clinical development 32 (3) (2024)
Sereirath Soth, Mikako Takakura, Masahiro Suekawa, Takayuki Onishi, Kiichi Hirohata, Tamami Hashimoto, Takahiro Maruno, Mitsuko Fukuhara, Yasuo Tsunaka, Tetsuo Torisu, Susumu Uchiyama
Quantification of full and empty particles of adeno-associated virus vectors via a novel dual fluorescence-linked immunosorbent assay. Molecular therapy. Methods & clinical development 32 (3) (2024)
Yoshiki Nakashima, Masayoshi Tsukahara
Atelocollagen supports three-dimensional culture of human induced pluripotent stem cells. Molecular therapy. Methods & clinical development 32 (3) (2024)
Savanna Skeeters, Kamal Bagale, Galina Stepanyuk, David Thieker, Aaron Aguhob, Kui K Chan, Benjamin Dutzar, Sergei Shalygin, Asif Shajahan, Xu Yang, Paul A DaRosa, Emily Frazier, Maximilian M Sauer, Lisa Bogatzki, Kelly A Byrnes-Blake, Yifan Song, Parastoo Azadi, Eric Tarcha, Lianghui Zhang, Erik Procko
Modulation of the pharmacokinetics of soluble ACE2 decoy receptors through glycosylation. Molecular therapy. Methods & clinical development 32 (3) (2024)
Noah K Taylor, Matthew J Guggenbiller, Pranali P Mistry, Oliver D King, Scott Q Harper
A self-complementary AAV proviral plasmid that reduces cross-packaging and ITR promoter activity in AAV vector preparations. Molecular therapy. Methods & clinical development 32 (3) (2024)
Xueyuan Liu, Riffard Jean-Gilles, Julia Baginski, Christina Cai, Ruilan Yan, Lili Zhang, Kevin Lance, Johannes C M van der Loo, Beverly L Davidson
Evaluation of a rapid multi-attribute combinatorial high-throughput UV-Vis/DLS/SLS analytical platform for rAAV quantification and characterization. Molecular therapy. Methods & clinical development 32 (3) (2024)
Yasuyuki Osanai, Yao Lulu Xing, Shinya Mochizuki, Kenta Kobayashi, Jihane Homman-Ludiye, Amali Cooray, Jasmine Poh, Ayumu Inutsuka, Nobuhiko Ohno, Tobias D Merson
5' Transgenes drive leaky expression of 3' transgenes in Cre-inducible bi-cistronic vectors. Molecular therapy. Methods & clinical development 32 (3) (2024)
Eduard H T M Ebberink, Alisa Ruisinger, Markus Nuebel, Helena Meyer-Berg, Irene R S Ferreira, Marco Thomann, Albert J R Heck
Probing recombinant AAV capsid integrity and genome release after thermal stress by mass photometry. Molecular therapy. Methods & clinical development 32 (3) (2024)
Sereirath Soth, Mikako Takakura, Masahiro Suekawa, Takayuki Onishi, Kiichi Hirohata, Tamami Hashimoto, Takahiro Maruno, Mitsuko Fukuhara, Yasuo Tsunaka, Tetsuo Torisu, Susumu Uchiyama
Quantification of full and empty particles of adeno-associated virus vectors via a novel dual fluorescence-linked immunosorbent assay. Molecular therapy. Methods & clinical development 32 (3) (2024)
Daima Bukini, Julie Makani, Joseph McCune, Dennis Lee, Cathy Bansbach, Serena De Vita, Dominic Kemps, Elianna Amin, Jonathan Spector, John Tisdale
Consensus-driven target product profiles for curative sickle cell disease gene therapies. Molecular therapy. Methods & clinical development 32 (3) (2024)
Alok Tanala Patra, Evan Tan, Yee Jiun Kok, Say Kong Ng, Xuezhi Bi
Temporal insights into molecular and cellular responses during rAAV production in HEK293T cells. Molecular therapy. Methods & clinical development 32 (3) (2024)
Shin Jeon, Jaein Park, Shibi Likhite, Ji Hwan Moon, Dongjun Shin, Liwen Li, Kathrin C Meyer, Jae W Lee, Soo-Kyung Lee
The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome. Molecular therapy. Methods & clinical development 32 (3) (2024)
Alok Tanala Patra, Evan Tan, Yee Jiun Kok, Say Kong Ng, Xuezhi Bi
Temporal insights into molecular and cellular responses during rAAV production in HEK293T cells. Molecular therapy. Methods & clinical development 32 (3) (2024)
Gina Blahetek, Christine Mayer, Johannes Zuber, Martin Lenter, Benjamin Strobel
Suppression of toxic transgene expression by optimized artificial miRNAs increases AAV vector yields in HEK-293 cells. Molecular therapy. Methods & clinical development 32 (3) (2024)
Amit Chhabra, George Bashirians, Christos J Petropoulos, Terri Wrin, Yuvika Paliwal, Peter V Henstock, Suryanarayan Somanathan, Candida da Fonseca Pereira, Ian Winburn, John E J Rasko
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies. Molecular therapy. Methods & clinical development 32 (3) (2024)
Gina Blahetek, Christine Mayer, Johannes Zuber, Martin Lenter, Benjamin Strobel
Suppression of toxic transgene expression by optimized artificial miRNAs increases AAV vector yields in HEK-293 cells. Molecular therapy. Methods & clinical development 32 (3) (2024)
Ada Irmak Özcan, Arianexys Aquino Lόpez, Alexandra N Wolff, Audrey Ma, Amanda Rosewell Shaw, Masataka Suzuki, Malcolm K Brenner, Mary K McKenna
Mesenchymal stromal cells protect combined oncolytic and helper-dependent adenoviruses from humoral immunity. Molecular therapy. Methods & clinical development 32 (3) (2024)
Lei Yu, Yong Zhou, Xin-Chang Shi, Guang-Yu Wang, Zhi-Hao Fu, Cheng-Gang Liang, Jun-Zhi Wang
An amplification-free CRISPR-Cas12a assay for titer determination and composition analysis of the rAAV genome. Molecular therapy. Methods & clinical development 32 (3) (2024)
Lei Yu, Yong Zhou, Xin-Chang Shi, Guang-Yu Wang, Zhi-Hao Fu, Cheng-Gang Liang, Jun-Zhi Wang
An amplification-free CRISPR-Cas12a assay for titer determination and composition analysis of the rAAV genome. Molecular therapy. Methods & clinical development 32 (3) (2024)
Savanna Skeeters, Kamal Bagale, Galina Stepanyuk, David Thieker, Aaron Aguhob, Kui K Chan, Benjamin Dutzar, Sergei Shalygin, Asif Shajahan, Xu Yang, Paul A DaRosa, Emily Frazier, Maximilian M Sauer, Lisa Bogatzki, Kelly A Byrnes-Blake, Yifan Song, Parastoo Azadi, Eric Tarcha, Lianghui Zhang, Erik Procko
Modulation of the pharmacokinetics of soluble ACE2 decoy receptors through glycosylation. Molecular therapy. Methods & clinical development 32 (3) (2024)
Daima Bukini, Julie Makani, Joseph McCune, Dennis Lee, Cathy Bansbach, Serena De Vita, Dominic Kemps, Elianna Amin, Jonathan Spector, John Tisdale
Consensus-driven target product profiles for curative sickle cell disease gene therapies. Molecular therapy. Methods & clinical development 32 (3) (2024)
Noah K Taylor, Matthew J Guggenbiller, Pranali P Mistry, Oliver D King, Scott Q Harper
A self-complementary AAV proviral plasmid that reduces cross-packaging and ITR promoter activity in AAV vector preparations. Molecular therapy. Methods & clinical development 32 (3) (2024)
Xueyuan Liu, Riffard Jean-Gilles, Julia Baginski, Christina Cai, Ruilan Yan, Lili Zhang, Kevin Lance, Johannes C M van der Loo, Beverly L Davidson
Evaluation of a rapid multi-attribute combinatorial high-throughput UV-Vis/DLS/SLS analytical platform for rAAV quantification and characterization. Molecular therapy. Methods & clinical development 32 (3) (2024)
Hikari Okada, Takeharu Sakamoto, Kouki Nio, Yingyi Li, Kazuyuki Kuroki, Saiho Sugimoto, Tetsuro Shimakami, Nobuhide Doi, Masao Honda, Motoharu Seiki, Shuichi Kaneko, Taro Yamashita
Lipid nanoparticle-encapsulated DOCK11-siRNA efficiently reduces hepatitis B virus cccDNA level in infected mice. Molecular therapy. Methods & clinical development 32 (3) (2024)
Noah K Taylor, Matthew J Guggenbiller, Pranali P Mistry, Oliver D King, Scott Q Harper
A self-complementary AAV proviral plasmid that reduces cross-packaging and ITR promoter activity in AAV vector preparations. Molecular therapy. Methods & clinical development 32 (3) (2024)
Daima Bukini, Julie Makani, Joseph McCune, Dennis Lee, Cathy Bansbach, Serena De Vita, Dominic Kemps, Elianna Amin, Jonathan Spector, John Tisdale
Consensus-driven target product profiles for curative sickle cell disease gene therapies. Molecular therapy. Methods & clinical development 32 (3) (2024)
Ashrafali Mohamed Ismail, Evan Witt, Taren Bouwman, Wyatt Clark, Bridget Yates, Matteo Franco, Sylvia Fong
The longitudinal kinetics of AAV5 vector integration profiles and evaluation of clonal expansion in mice. Molecular therapy. Methods & clinical development 32 (3) (2024)
Eduard H T M Ebberink, Alisa Ruisinger, Markus Nuebel, Helena Meyer-Berg, Irene R S Ferreira, Marco Thomann, Albert J R Heck
Probing recombinant AAV capsid integrity and genome release after thermal stress by mass photometry. Molecular therapy. Methods & clinical development 32 (3) (2024)
Yasuyuki Osanai, Yao Lulu Xing, Shinya Mochizuki, Kenta Kobayashi, Jihane Homman-Ludiye, Amali Cooray, Jasmine Poh, Ayumu Inutsuka, Nobuhiko Ohno, Tobias D Merson
5' Transgenes drive leaky expression of 3' transgenes in Cre-inducible bi-cistronic vectors. Molecular therapy. Methods & clinical development 32 (3) (2024)
Robert D E Clark, Felix Rabito, Ferris T Munyonho, T Parks Remcho, Jay K Kolls
Evaluation of anti-vector immune responses to adenovirus-mediated lung gene therapy and modulation by αCD20. Molecular therapy. Methods & clinical development 32 (3) (2024)
Ramya Rallabandi, Brenna Sharp, Spencer Majerus, Austin Royster, Sarrianna Hoffer, Mia Ikeda, Patricia Devaux
Engineering single-cycle MeV vector for CRISPR-Cas9 gene editing. Molecular therapy. Methods & clinical development 32 (3) (2024)
Robert D E Clark, Felix Rabito, Ferris T Munyonho, T Parks Remcho, Jay K Kolls
Evaluation of anti-vector immune responses to adenovirus-mediated lung gene therapy and modulation by αCD20. Molecular therapy. Methods & clinical development 32 (3) (2024)
Savanna Skeeters, Kamal Bagale, Galina Stepanyuk, David Thieker, Aaron Aguhob, Kui K Chan, Benjamin Dutzar, Sergei Shalygin, Asif Shajahan, Xu Yang, Paul A DaRosa, Emily Frazier, Maximilian M Sauer, Lisa Bogatzki, Kelly A Byrnes-Blake, Yifan Song, Parastoo Azadi, Eric Tarcha, Lianghui Zhang, Erik Procko
Modulation of the pharmacokinetics of soluble ACE2 decoy receptors through glycosylation. Molecular therapy. Methods & clinical development 32 (3) (2024)
Amit Chhabra, George Bashirians, Christos J Petropoulos, Terri Wrin, Yuvika Paliwal, Peter V Henstock, Suryanarayan Somanathan, Candida da Fonseca Pereira, Ian Winburn, John E J Rasko
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies. Molecular therapy. Methods & clinical development 32 (3) (2024)
Shin Jeon, Jaein Park, Shibi Likhite, Ji Hwan Moon, Dongjun Shin, Liwen Li, Kathrin C Meyer, Jae W Lee, Soo-Kyung Lee
The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome. Molecular therapy. Methods & clinical development 32 (3) (2024)
Anna Mateu-Bosch, Eulàlia Segur-Bailach, Emma Muñoz-Moreno, María José Barallobre, Maria Lourdes Arbonés, Sabrina Gea-Sorlí, Frederic Tort, Antonia Ribes, Judit García-Villoria, Cristina Fillat
Systemic delivery of AAV-GCDH ameliorates HLD-induced phenotype in a glutaric aciduria type I mouse model. Molecular therapy. Methods & clinical development 32 (3) (2024)
Davindra Singh, Elahe Memari, Stephanie He, Hossein Yusefi, Brandon Helfield
Cardiac gene delivery using ultrasound: State of the field. Molecular therapy. Methods & clinical development 32 (3) (2024)
Anna Mateu-Bosch, Eulàlia Segur-Bailach, Emma Muñoz-Moreno, María José Barallobre, Maria Lourdes Arbonés, Sabrina Gea-Sorlí, Frederic Tort, Antonia Ribes, Judit García-Villoria, Cristina Fillat
Systemic delivery of AAV-GCDH ameliorates HLD-induced phenotype in a glutaric aciduria type I mouse model. Molecular therapy. Methods & clinical development 32 (3) (2024)
Dongxin Wang, Dongsheng Duan
Humoral and cellular immune responses to AAV delivery in the airway. Molecular therapy. Methods & clinical development 32 (3) (2024)
Esther Osher, Yossi Anis, Ruth Singer-Shapiro, Nataly Urshanski, Tamar Unger, Shira Albeck, Oren Bogin, Gary Weisinger, Fortune Kohen, Avi Valevski, Aviva Fattal-Valevski, Liora Sagi, Michal Weitman, Yulia Shenberger, Nadav Sagiv, Ruth Navon, Meir Wilchek, Naftali Stern
Treating late-onset Tay Sachs disease: Brain delivery with a dual trojan horse protein. Molecular therapy. Methods & clinical development 32 (3) (2024)
Carly M Bliss, Sarah L Hulin-Curtis, Marta Williams, Mahulena Marušková, James A Davies, Evelina Statkute, Alexander T Baker, Louise Stack, Lucas Kerstetter, Lauren E Kerr-Jones, Kate F Milward, Gabrielle Russell, Sarah J George, Luned M Badder, Richard J Stanton, Lynda Coughlan, Ian R Humphreys, Alan L Parker
A pseudotyped adenovirus serotype 5 vector with serotype 49 fiber knob is an effective vector for vaccine and gene therapy applications. Molecular therapy. Methods & clinical development 32 (3) (2024)

volume 32, number 4, 2024

Thomas Sécher, Mélanie Cortes, Chloé Boisseau, Marie-Thérèse Barba Goudiaby, Aubin Pitiot, Christelle Parent, Muriel Thomas, Nathalie Heuzé-Vourc'h
Synergy between Lactobacillus murinus and anti-PcrV antibody delivered in the airways to boost protection against Pseudomonas aeruginosa . Molecular therapy. Methods & clinical development 32 (4) (2024)
Paul G Ayoub, Julia Gensheimer, Lindsay Lathrop, Colin Juett, Jason Quintos, Kevin Tam, Jack Reid, Feiyang Ma, Curtis Tam, Grace E McAuley, Devin Brown, Xiaomeng Wu, Ruixue Zhang, Kathryn Bradford, Roger P Hollis, Gay M Crooks, Donald B Kohn
Lentiviral vectors for precise expression to treat X-linked lymphoproliferative disease. Molecular therapy. Methods & clinical development 32 (4) (2024)
Adrian Schimek, Judy K M Ng, Ioannes Basbas, Fabian Martin, Dongyue Xin, David Saleh, Jürgen Hubbuch
Erratum: An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development. Molecular therapy. Methods & clinical development 32 (4) (2024)
Konstantina Tzimou, David Catalán-Tatjer, Lars K Nielsen, Jesús Lavado-García
Unlocking DOE potential by selecting the most appropriate design for rAAV optimization. Molecular therapy. Methods & clinical development 32 (4) (2024)