Results 141 to 150 of about 12,260,520 (401)

iPSC-Derived Intestinal Organoids from Cystic Fibrosis Patients Acquire CFTR Activity upon TALEN-Mediated Repair of the p.F508del Mutation

Molecular Therapy: Methods & Clinical Development, 2020
Cystic fibrosis (CF) is the main genetic cause of death among the Caucasian population. The disease is characterized by abnormal fluid and electrolyte mobility across secretory epithelia.
Aarne Fleischer, Sara Vallejo-Díez, José María Martín-Fernández, Almudena Sánchez-Gilabert, Mónica Castresana, Angel del Pozo, Amaia Esquisabel, Silvia Ávila, José Luis Castrillo, Eusebio Gaínza, José Luis Pedraz, Miguel Viñas, Daniel Bachiller   +12 more
doaj  

Targeting the Apoa1 locus for liver-directed gene therapy

Molecular Therapy: Methods & Clinical Development, 2021
Clinical application of somatic genome editing requires therapeutics that are generalizable to a broad range of patients. Targeted insertion of promoterless transgenes can ensure that edits are permanent and broadly applicable while minimizing risks of ...
Marco De Giorgi, Ang Li, Ayrea Hurley, Mercedes Barzi, Alexandria M. Doerfler, Nikitha A. Cherayil, Harrison E. Smith, Jonathan D. Brown, Charles Y. Lin, Karl-Dimiter Bissig, Gang Bao, William R. Lagor   +11 more
doaj  

Efficient gene targeting by TAL effector nucleases coinjected with exonucleases in zygotes

Scientific Reports, 2013
TAL Effector Nucleases (TALENs) are versatile tools for targeted gene editing in various species. However, their efficiency is still insufficient, especially in mammalian embryos.
T. Mashimo, T. Kaneko, Tetsushi Sakuma, J. Kobayashi, Y. Kunihiro, B. Voigt, Takashi Yamamoto, T. Serikawa   +7 more
semanticscholar   +1 more source

Mechanisms and kinetic assays of aminoacyl‐tRNA synthetases

FEBS Letters, EarlyView.
Igor Zivkovic, Morana Dulic, Ita Gruic‐Sovulj   +2 more
wiley   +1 more source

MET variants with activating N‐lobe mutations identified in hereditary papillary renal cell carcinomas still require ligand stimulation

Molecular Oncology, EarlyView.
MET variants in the N‐lobe of the kinase domain, found in hereditary papillary renal cell carcinoma, require ligand stimulation to promote cell transformation, in contrast to other RTK variants. This suggests that HGF expression in the microenvironment is important for tumor growth in such patients. Their sensitivity to MET inhibitors opens the way for
Célia Guérin, Audrey Vinchent, Marie Fernandes, Isabelle Damour, Agathe Laratte, Rémi Tellier, Gabriella O. Estevam, Jean‐Pascal Meneboo, Céline Villenet, Clotilde Descarpentries, James S. Fraser, Martin Figeac, Alexis B. Cortot, Etienne Rouleau, David Tulasne   +14 more
wiley   +1 more source

CRISPR/Cascade 9-Mediated Genome Editing-Challenges and Opportunities

Frontiers in Genetics, 2018
Clustered Regularly Interspaced Palindromic Repeats (CRISPR) and Cascade 9 (also known as Cas9, CRISPR associated protein 9) confer protection against invading viruses or plasmids.
Bhaskar Roy, Jing Zhao, Jing Zhao, Chao Yang, Wen Luo, Teng Xiong, Yong Li, Xiaodong Fang, Guanjun Gao, Chabungbam O. Singh, Lise Madsen, Lise Madsen, Lise Madsen, Yong Zhou, Karsten Kristiansen, Karsten Kristiansen   +15 more
doaj   +1 more source

A Targetron System for Gene Targeting in Thermophiles and Its Application in Clostridium thermocellum

PLoS ONE, 2013
Background Targetrons are gene targeting vectors derived from mobile group II introns. They consist of an autocatalytic intron RNA (a “ribozyme”) and an intron-encoded reverse transcriptase, which use their combined activities to achieve highly efficient
Georg Mohr, Wei Hong, Jie Zhang, Gu-zhen Cui, Yunfeng Yang, Q. Cui, Ya-Jun Liu, A. Lambowitz   +7 more
semanticscholar   +1 more source

Peripheral blood proteome biomarkers distinguish immunosuppressive features of cancer progression

Molecular Oncology, EarlyView.
Immune status significantly influences cancer progression. This study used plasma proteomics to analyze benign 67NR and malignant 4T1 breast tumor models at early and late tumor stages. Immune‐related proteins–osteopontin (Spp1), lactotransferrin (Ltf), calreticulin (Calr) and peroxiredoxin 2 (Prdx2)–were associated with systemic myeloid‐derived ...
Yeon Ji Park, Jae Won Oh, Hyewon Chung, Jung Won Kwon, Yi Rang Na, Kwang Pyo Kim, Seung Hyeok Seok   +6 more
wiley   +1 more source

RNase P Ribozymes Inhibit the Replication of Human Cytomegalovirus by Targeting Essential Viral Capsid Proteins

Viruses, 2015
An engineered RNase P-based ribozyme variant, which was generated using the in vitro selection procedure, was used to target the overlapping mRNA region of two proteins essential for human cytomegalovirus (HCMV) replication: capsid assembly protein (AP ...
Zhu Yang, Michael Reeves, Jun Ye, Phong Trang, Li Zhu, Jingxue Sheng, Yu Wang, Ke Zen, Jianguo Wu, Fenyong Liu   +9 more
doaj   +1 more source

Autoimmune T lymphocytes in myasthenia gravis. Determination of target epitopes using T lines and recombinant products of the mouse nicotinic acetylcholine receptor gene. [PDF]

, 1989
Arthur Melms, Susanne Chrestel, Berthold Schalke, Hartmut Wekerle, Alex Mauron, Marc Ballivet, T. Barkas   +6 more
openalex   +1 more source