Results 51 to 60 of about 45,101 (293)

Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector

Molecular Therapy: Methods & Clinical Development, 2017
Wiskott-Aldrich syndrome (WAS) is a life-threatening immunodeficiency caused by mutations within the WAS gene. Viral gene therapy to restore WAS protein (WASp) expression in hematopoietic cells of patients with WAS has the potential to improve outcomes ...
Swati Singh, Iram Khan, Socheath Khim, Brenda Seymour, Karen Sommer, Matthew Wielgosz, Zachary Norgaard, Hans-Peter Kiem, Jennifer Adair, Denny Liggitt, Arthur Nienhuis, David J. Rawlings   +11 more
doaj   +1 more source

Germline transgenesis and insertional mutagenesis in the ascidian Ciona intestinalis [PDF]

, 2007
Stable transgenesis is a splendid technique that is applicable to the creation of useful marker lines,enhancer/gene traps, and insertional mutagenesis.
Sasakura Yasunori, 笹倉 靖徳
core   +1 more source

Cracking the Code: Genotype–Phenotype Correlation Models in Sarcoglycanopathies

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Sarcoglycanopathies are among the most severe limb‐girdle muscular dystrophies (LGMD), though milder presentations have been described. These diseases are primarily caused by missense variants, but the limited predictability of their effect on protein maturation, complex formation, and transport has hindered reliable genotype ...
Leonela Luce, Goknur Selen Kocak, José Verdú‐Díaz, Jorge Alonso‐Pérez, Kristl G. Claeys, Tanya Stojkovic, Gorka Fernández‐Eulate, Pascal Laforêt, Najoua Miladi, Filipe Di Pace, Cristiane Araujo Martins Moreno, Edmar Zanoteli, Conrad C. Weihl, Volker Straub, Ana Töpf, Jordi Díaz‐Manera, Sarcoglycan European Cohort Consortium, Adele D′Amico, Adolfo López de Munain, Alicia Alonso‐Jiménez, Ana Camacho‐Salas, Andrea Gangfuß, Andrés Nascimento, Anna Sarkozy, Anneke J. van der Kooi, Arturo Fraga‐Bau, Béla Melegh, Benedikt Schoser, Bjarne Udd, Blaz Koritnik, Carlos Ortez, Chiara Marini Bettolo, Chiara Panicucci, Claudia Weiss, Claudio Bruno, Claudio Semplicini, Cristina Dominguez‐González, Cristina Garrido, David Gómez‐Andrés, Edoardo Malfatti, Elena Pegoraro, Elke De Vos, Francina Munell, Gabriele Dekomien, Giacomo Pietro Comi, Giorgio Tasca, Isabelle Richard, Jan L. De Bleecker, Jana Haberlová, Jesper Helbo Storgaard, Johanna Palmio, John Vissing, Juan Carlos de Leon‐Hernández, Kinga Hadzsiev, Laura Costa‐Comellas, Lea Leonardis, Leroy ten Dam, Lidia González‐Quereda, Luca Bello, Luisa Politano, Manuela Santos, Marianne de Visser, Marie Rohlenová, Matteo Garibaldi, Michela Guglieri, Nicolas Deconinck, Nicoline Løkken, Omar Abdel‐Mannan, Pia Gallano, Roberto Fernández‐Torrón, Ulrike Schara‐Schmidt, Vincenzo Nigro, Vittoria Zangaro   +72 more
wiley   +1 more source

Genome-wide transposon mutagenesis of paramyxoviruses reveals constraints on genomic plasticity.

PLoS Pathogens, 2020
The antigenic and genomic stability of paramyxoviruses remains a mystery. Here, we evaluate the genetic plasticity of Sendai virus (SeV) and mumps virus (MuV), sialic acid-using paramyxoviruses that infect mammals from two Paramyxoviridae subfamilies ...
Satoshi Ikegame, Shannon M Beaty, Christian Stevens, Taylor Won, Arnold Park, David Sachs, Patrick Hong, Benhur Lee, Patricia A Thibault   +8 more
doaj   +1 more source

ApuA, a multifunctional x-glucan-degrading enzyme of Streptococcus suis, mediates adhesion to porcine epithelium and mucus [PDF]

, 2010
We have identified apuA in Streptococcus suis, which encodes a bifunctional amylopullulanase with conserved -amylase and pullulanase substrate-binding domains and catalytic motifs.
Ferrando, M.L., Fuentes, S., Greeff, A., de, Smith, H.E., Wells, J.   +4 more
core   +2 more sources

Microfluidic Hydroporation Platform for Effective Allogeneic CAR‐T Cell Production and Subsequent Functional Cytotoxicity Analysis

Advanced Materials Technologies, EarlyView.
The hydroporator platform employs controlled hydrodynamic deformation for efficient mRNA and CRISPR/Cas9 delivery into primary human T cells, enabling allogeneic CAR‐T cell manufacturing. It preserves cell functionality and drives potent gene editing, CAR expression, and tumor cytotoxicity, while feature‐based analysis links these functional outcomes ...
Soohyun Jeon, Sein Kim, Hyun‐Young Kim, Duck Cho, Sungsu Park, Seohyun Kim, Aram J. Chung   +6 more
wiley   +1 more source

In vitro HIV DNA integration in STAT3 drives T cell persistence-A model of HIV-associated T cell lymphoma.

PLoS Pathogens
Oncogenic retroviruses are known for their pathogenesis via insertional mutagenesis, in which the presence of a provirus and its transcriptional control elements alter the expression of a nearby or surrounding host gene.
Michael Rist, Machika Kaku, John M Coffin   +2 more
doaj   +1 more source

General and Specific Promotion of Flagellar Assembly by a Flagellar Nucleoside Diphosphate Kinase [PDF]

, 2017
Nucleoside diphosphate kinases (NDKs) play a central role in diverse cellular processes using the canonical NDK activity or alternative mechanisms that remain poorly defined.
Ciruelas, Kristine S., Diener, Dennis R., Gopal, Radhika, Ishikawa, Takashi, King, Stephen M., Liu, Yi, Maizy, Yousif, Poghosyan, Emiliya, Yang, Pinfen, Zhu, Xiaoyan   +9 more
core   +1 more source

The use of retroviral vectors for gene therapy-what are the risks? A review of retroviral pathogenesis and its relevance to retroviral vector-mediated gene delivery [PDF]

, 2004
© 2004 Anson; licensee BioMed Central Ltd. This is an open-access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction
Donald S Anson
core   +1 more source

Testicular mRNA‐LNP Delivery: A Novel Therapy for Genetic Spermatogenic Disorders

Advanced Science, EarlyView.
In the current study, we developed a novel therapy for genetic spermatogenic disorders. It was revealed that in vivo delivery of mRNA LNP3 could restore spermatogenesis in Msh5D486Y/D486Y and Maps KO mouse models with meiotic arrest. Notably, the offspring without genomic integration was born using ICSI derived from the rescue of Msh5D486Y/D486Y mouse ...
Chenwang Zhang, Nan Liang, Wenbo Li, Shuai Xu, Peng Li, Wanze Ni, Na Li, Sha Han, Ningjing Ou, Haowei Bai, Yuxiang Zhang, Furong Bai, Yifan Sun, Dewei Qian, Xinjie Bu, Erlei Zhi, Ruhui Tian, Yuhua Huang, Jingpeng Zhao, Fujun Zhao, Hao Chen, Zheng Li, Chencheng Yao   +22 more
wiley   +1 more source