Results 171 to 180 of about 1,217,743 (262)

Advances in Gene Therapy for Inherited Haemoglobinopathies. [PDF]

Hematol Rep
Gaspar AB, Gaspar HB.
europepmc   +1 more source

Iron homeostasis disruption and lipid peroxidation in skeletal muscle during short‐term immobilization

FEBS Open Bio, EarlyView.
14‐day casting‐induced immobilization reduced gastrocnemius muscle mass and increased non‐heme iron and ferritin heavy chain levels. Despite iron accumulation, transferrin receptor 1 and iron regulatory protein 2 were paradoxically upregulated. Lipid peroxidation was elevated without compensatory antioxidant responses.
Haruka Yokogawa, Kazuhiko Higashida, Naoya Nakai   +2 more
wiley   +1 more source

Advances in Gene Therapy for Age-Related Macular Degeneration: A Narrative Review. [PDF]

J Clin Med
Ahmad NU, Mir TA.
europepmc   +1 more source

Proteasomal degradation of intracellularly expressed Amblyomin‐X limits suicide gene therapy potential in melanoma cells

FEBS Open Bio, EarlyView.
This study explores the feasibility of expressing the antitumoral protein Amblyomin‐X through a suicide gene therapy approach and investigates its intracellular fate after gene delivery. Although the gene is efficiently expressed, melanoma cells rapidly degrade the Amblyomin‐X protein via proteasome activity.
Victor Dal Posolo Cinel, Carlos DeOcesano Pereira, Aline Ramos Maia Lobba, Melissa Regina Fessel, Ana Marisa Chudzinski‐Tavassi   +4 more
wiley   +1 more source

Enhancer-based gene therapy: a new path for precision medicine. [PDF]

Hereditas
Marei HE.
europepmc   +1 more source

P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up*

Genetics in Medicine Open
Francesca Fumagalli, Valeria Calbi, Fabiola De Mattia, Alberto Zambon, Vera Gallo, Salvatore Recupero, Elena Sophia Fratini, Alessia Ippolito, Francesca Ciotti, Maddalena Fraschini, Marina Sarzana, Stefano Scarparo, Eugenio Montini, Sara Locatelli, Marcella Facchini, Alessandra Clerici, Francesco Morena, Sabata Martino, Muska Yarzi, Sean Moro, Andrew Shenker, Jean Brooks, Alan Richardson, Leslie Meltzer, Laura Campbell, Alessandro Aiuti   +25 more
doaj   +1 more source

MiR‐513a promotes human erythroid differentiation by modulating c‐Jun

FEBS Open Bio, EarlyView.
During early human erythropoiesis, miR‐513a promoted erythroid differentiation in primary human CD34+ hematopoietic stem‐progenitor cells and human TF‐1 erythroleukemic cells by indirectly decreasing c‐Jun and phospho‐c‐Jun expression, which are associated with increased GATA1 expression.
MinJung Kim, Brittany Taylor, Shannon Bolten, Christian L. Eberly, Mahliya Abdurahman, T. Michael Creed, Acong Yang, Taylor L. Hatchet, Tristan Dyson, Shuo Gu, Curt I. Civin, Tami J. Kingsbury   +11 more
wiley   +1 more source

Bridging science and hope: the evolving story of gene therapy for neuromuscular diseases. [PDF]

Front Cell Dev Biol
Wein N, Barthélémy F.
europepmc   +1 more source

Rapid screening of staphylokinase protein variants using an unpurified cell‐free expression system

FEBS Open Bio, EarlyView.
An unpurified cell‐free protein synthesis (CFPS) platform enables rapid functional screening of staphylokinase variants. Direct plasminogen‐activation assays performed in microplate format provide real‐time activity readouts, allowing rapid identification and ranking of variants with improved or reduced fibrinolytic activity without protein ...
Maria Tomková, Veronika Hovanová, Jiri Damborsky, Erik Sedlák   +3 more
wiley   +1 more source