Results 61 to 70 of about 172,174 (293)

In Vivo and Ex Vivo Gene Electrotransfer in Ophthalmological Disorders

, 2022
The aim of this document is to present an overview of gene electrotransfer in ophthalmological disorders. In order to ensure an adequate variety of the assessed studies, several electronic databases were considered and studies published between January ...
Roberta Fusco, Perazzolo Gallo, Giacomo, Ruggero Cadossi, Valeria D’Alessio, Mattia Ronchetti, D’Alessio, Valeria, Elio Di Bernardo, Cadossi, Matteo, Ronchetti, Mattia, Fusco, Roberta, Di Bernardo, Elio, Cadossi, Ruggero, Matteo Cadossi, Giacomo Perazzolo Gallo   +13 more
core   +1 more source

From mice to humans—divergent strategies for intestinal homeostasis and regeneration

FEBS Letters, EarlyView.
Recent advances such as organoid genome editing, xenotransplantation, imaging, and whole‐genome sequencing have enabled direct studies of human intestinal stem cells (ISCs). These studies reveal species‐specific features, including slower ISC proliferation, distinct injury responses, slower somatic mutation accumulation in humans, and an inverse ...
Keiko Ishikawa, Shinya Sugimoto, Toshiro Sato   +2 more
wiley   +1 more source

New Frontiers of Corneal Gene Therapy [PDF]

, 2019
Corneal diseases are among the most prevalent causes of blindness worldwide. The transparency and clarity of the cornea are guaranteed by a delicate physiological, anatomic, and functional balance.
Ghassabian, Hanieh, Barbaro, Vanessa, Di Iorio, Enzo, Ferrari, Stefano, Alvisi, Gualtiero, Trevisan, Marta, Masi, Giulia, Ponzin, Diego, Nespeca, Patrizia, Palù, Giorgio   +9 more
core   +1 more source

Motor and neurocognitive profiles of children with symptomatic spinal muscular atrophy type 1 with two copies of SMN2 before and after treatment: a longitudinal observational study

Frontiers in Neurology
IntroductionSpinal muscular atrophy (SMA) is a neurodegenerative disease caused by mutations in the survival motor neuron 1 (SMN1) gene. In clinical studies, gene replacement therapy with onasemnogene abeparvovec (formerly AVXS-101, Zolgensma®, Novartis)
Ilaria Bitetti, Maria Rosaria Manna, Roberto Stella, Antonio Varone   +3 more
doaj   +1 more source

Structural insights and therapeutic targets in Acinetobacter baumannii capsule biosynthesis

FEBS Letters, EarlyView.
Hypervirulent KL49 A. baumannii's capsular polysaccharide contains the nonulosonic acid 8‐epi‐Leg5,7Ac2, synthesized by epimerization via ElaA, ElaB, and ElaC. Crystal structures of ElaA, ElaB, and ElaC reveal their role in CMP‐Leg5,7Ac2 synthesis and regioselective C8 epimerization.
Woo Cheol Lee, Junho Jeong, Chae Yeong Lee, Hyunjoon Cho, Man Hwan Oh, Je Chul Lee, Jee‐Young Lee, Yangmee Kim   +7 more
wiley   +1 more source

The magnetofection method: Using magnetic force to enhance gene delivery [PDF]

, 2003
In order to enhance and target gene delivery we have previously established a novel method, termed magnetofection, which uses magnetic force acting on gene vectors that are associated with magnetic particles.
Remy, J. (Jean-Serge), Lausier, J. (Jim), Schillinger, U. (Ulrike), Scherer, F., Rosenecker, J., Anton, M. (Marina), Plank, C., Schillinger, U., Rémy, J.-S., Rosenecker, J. (Joseph), Krötz, F. (Florian), Lausier, J., Scherer, F. (Franz), Bergemann, C., Plank, C. (Christian), Anton, M., Krötz, F., Bergemann, C. (Christian)   +17 more
core   +1 more source

The role of miR‐335‐5p in the redifferentiation of BRAF p.V600E thyroid cancers

Molecular Oncology, EarlyView.
The BRAF p.V600E mutation promotes thyroid cancer dedifferentiation and radioiodine resistance. Using a network approach, we identified miR‐335‐5p as a key regulator of BRAF‐mutated thyroid tumors. Restoring miR‐335‐5p increased thyroid‐specific gene expression and iodine uptake in cells and organoids.
Valeria Pecce, Simone Bini, Marialuisa Sponziello, Giorgio Grani, Giulia Fiscon, Paola Paci, Lorenzo Farina, Rosa Falcone, Valentina Maggisano, Sebastiano Filetti, Cosimo Durante, Antonella Verrienti   +11 more
wiley   +1 more source

Current status of gene therapy for alpha-1 antitrypsin deficiency

, 2015
INTRODUCTION: As a common monogenic disease, alpha-1 antitrypsin (AAT) deficiency has undergone thorough investigation for the development of gene therapy.
Flotte, Terence R., Loring, Heather S.
core   +1 more source

Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators

, 2023
Familial dysautonomia (FD) is a rare neurodevelopmental and neurodegenerative disease caused by a splicing mutation in the Elongator Acetyltransferase Complex Subunit 1 (ELP1) gene.
Chaverra, Marta, Punzo, Claudio, Zhao, Xin, Morini, Elisabetta, King, Colin, Weetall, Marla, Kirchner, Emily, Miettinen, Heini, Cheng, Shun-Yun, Lefcort, Frances, Slaugenhaupt, Susan, George, Lynn, Narasimhan, Jana, Costello, Stephanann, Schultz, Anastasia   +14 more
core   +1 more source

Dimethyl fumarate combined with cisplatin at subcytotoxic doses sensitizes cervical cancer toward ferroptosis and apoptosis through GSH restriction and p53 (re)activation

Molecular Oncology, EarlyView.
Dimethyl fumarate (DMF) reduces growth of HPV‐positive cervical cancer spheroids and induces ferroptosis in cervical cancer cells via blocking SLC7A11/Glutathione (GSH) axis. Combination of subcytotoxic doses of DMF and cisplatin (CDDP) further suppresses spheroid growth and drives cell death in 2D culture models.
Carolina Punziano, Giuseppina Minopoli, Simona Romano, Laura Marrone, Katia Aquilano, Maria Lina Tornesello, Raffaella Faraonio   +6 more
wiley   +1 more source