Zobrazeno 1 - 10
of 301
pro vyhledávání: '"MT: delivery strategies"'
Autor:
Qiuhan Sun, Fangzhi Tan, Xinlin Wang, Xingliang Gu, Xin Chen, Yicheng Lu, Nianci Li, Xiaoyun Qian, Yinyi Zhou, Ziyu Zhang, Man Wang, Liyan Zhang, Busheng Tong, Jieyu Qi, Renjie Chai
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102396- (2024)
Inner ear hair cell (HC) damage is irreversible in mammals, but it has been shown that supporting cells (SCs) have the potential to differentiate into HCs. Serpine2, a serine protease inhibitor, encodes protease nexin 1, and this has been suggested t
Externí odkaz:
https://doaj.org/article/b08deec598944730915cc540aac955cd
Autor:
Denise Klatt, Lucia Sereni, Boya Liu, Pietro Genovese, Axel Schambach, Els Verhoeyen, David A. Williams, Christian Brendel
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102389- (2024)
The baboon endogenous retrovirus (BaEV) glycoprotein is superior to the commonly used vesicular stomatitis virus glycoprotein (VSVg) for retroviral gene transfer into resting hematopoietic stem cells and lymphocyte populations. The derivative BaEVRLe
Externí odkaz:
https://doaj.org/article/8e2e01e0ba9340b482b868e06259719f
Autor:
Narumi Uno, Hitomaru Miyamoto, Kyotaro Yamazaki, Masaya Egawa, Hiroaki Kobayashi, Kanako Kazuki, Mitsuhiko Osaki, Teruhiko Suzuki, Shusei Hamamichi, Mitsuo Oshimura, Kazuma Tomizuka, Yasuhiro Kazuki
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102382- (2024)
Microcell-mediated chromosome transfer (MMCT) is anticipated as a unique strategy to manipulate numbers of chromosomes, including the generation of hyperaneuploidy syndrome models with human induced pluripotent stem cells (hiPSCs). Mouse A9/Chinese h
Externí odkaz:
https://doaj.org/article/319aaab124b44ea0aede57a005bc3f48
Autor:
Melissa R. Soto, Mae M. Lewis, Jasmim Leal, Yuting Pan, Rashmi P. Mohanty, Arian Veyssi, Esther Y. Maier, Brittany J. Heiser, Debadyuti Ghosh
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102375- (2024)
For cystic fibrosis patients, a lung-targeted gene therapy would significantly alleviate pulmonary complications associated with morbidity and mortality. However, mucus in the airways and cell entry pose huge delivery barriers for local gene therapy.
Externí odkaz:
https://doaj.org/article/e8f77c73d20b47b7862c329c25cf9246
Autor:
Pedro Justicia-Lirio, María Tristán-Manzano, Noelia Maldonado-Pérez, Carmen Barbero-Jiménez, Marina Cortijo-Gutiérrez, Kristina Pavlovic, Francisco J. Molina-Estevez, Pilar Muñoz, Ana Hinckley-Boned, Juan R. Rodriguez-Madoz, Felipe Prosper, Carmen Griñán-Lison, Saúl A. Navarro-Marchal, Carla Panisello, Julia Muñoz-Ballester, Pedro A. González-Sierra, Concha Herrera, Juan A. Marchal, Francisco Martín
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102308- (2024)
Although chimeric antigen receptor (CAR) T cell therapy has revolutionized type B cancer treatment, efficacy remains limited in various lymphomas and solid tumors. Reinforcing conventional CAR-T cells to release cytokines can improve their efficacy b
Externí odkaz:
https://doaj.org/article/bffb79b98bd04d24b3575e2ddc65fa95
Autor:
Masanori Sawamura, Kiyoshi Tachikawa, Rie Hikawa, Hisako Akiyama, Seiji Kaji, Ken Yasuda, Angel I. Leu, Hyojung Hong, Rajesh Mukthavaram, Pad Chivukula, Hodaka Yamakado, Yoshio Hirabayashi, Ryosuke Takahashi, Shu-ichi Matsuzawa
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102380- (2024)
Despite the wide range of applications of mRNA therapies, major difficulties exist in the efficient delivery of mRNA into oligodendrocytes, a type of glial cell in the brain. Commonly used viral vectors are not efficient in transforming oligodendrocy
Externí odkaz:
https://doaj.org/article/a690f55095224122b98cf44013d30ce2
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102373- (2024)
RNA science has experienced a surge, particularly its application to vaccine and therapeutic development. Delivery of RNA molecules into the brain remains one of the biggest therapeutic challenges with the blood-brain barrier excluding most drug-deli
Externí odkaz:
https://doaj.org/article/68adf42961f84a15be8123bd69a06270
Autor:
Angela L. Hughson, Gary Hannon, Noah A. Salama, Tara G. Vrooman, Caroline A. Stockwell, Bradley N. Mills, Jesse Garrett-Larsen, Haoming Qiu, Roula Katerji, Lauren Benoodt, Carl J. Johnston, Joseph D. Murphy, Emma Kruger, Jian Ye, Nicholas W. Gavras, David C. Keeley, Shuyang S. Qin, Maggie L. Lesch, Jason B. Muhitch, Tanzy M.T. Love, Laura M. Calvi, Edith M. Lord, Nadia Luheshi, Jim Elyes, David C. Linehan, Scott A. Gerber
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102350- (2024)
Pronounced T cell exhaustion characterizes immunosuppressive tumors, with the tumor microenvironment (TME) employing multiple mechanisms to elicit this suppression. Traditional immunotherapies, such as immune checkpoint blockade, often fail due to th
Externí odkaz:
https://doaj.org/article/f027f2b3c534440cbdc1ca41fe01b771
Autor:
Sonja Korhonen, Katja Stenberg, Umair Seemab, Piia Bartos, Katariina Mäkiniemi, Jørgen Kjems, Daniel Miotto Dupont, Astrid Subrizi
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102352- (2024)
Ocular diseases create a significant economic burden and decrease in quality of life worldwide. Drugs and carrier molecules that penetrate ocular tissues after intravenous administration are needed for more efficient and patient-friendly treatment of
Externí odkaz:
https://doaj.org/article/f5d64ecff7614091bfcc85a7b1d0e6be
Autor:
Juliette Pulman, Catherine Botto, Hugo Malki, Duohao Ren, Paul Oudin, Anne De Cian, Marie As, Charlotte Izabelle, Bruno Saubamea, Valerie Forster, Stéphane Fouquet, Camille Robert, Céline Portal, Aziz El-Amraoui, Sylvain Fisson, Jean-Paul Concordet, Deniz Dalkara
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102349- (2024)
Genome editing by CRISPR-Cas holds promise for the treatment of retinal dystrophies. For therapeutic gene editing, transient delivery of CRISPR-Cas9 is preferable to viral delivery which leads to long-term expression with potential adverse consequenc
Externí odkaz:
https://doaj.org/article/79b809bb916648639568bb98681ed82b