Results 51 to 60 of about 1,204,526 (212)

Gene therapy Progress and Prospects: Gene therapy for the hemophilias [PDF]

Gene Therapy, 2003
Recent gene transfer trials for hemophilia A and B, bleeding disorders lacking either functional factor VIII or IX, respectively, have produced tantalizing results, suggesting that the potential to correct these bleeding disorders at a molecular level may be at hand.
openaire   +2 more sources

P314: Long-term safety and sustained functional benefit in patients with DMD 4 years post-treatment with delandistrogene moxeparvovec: A phase 1/2a study*

Genetics in Medicine Open, 2023
Erin O’Rourke, Jerry R. Mendell, Zarife Sahenk, Kelly J. Lehman, Linda P. Lowes, Natalie F. Reash, Megan Iammarino, Lindsay Alfano, Sarah Lewis, Kathleen Church, Richard Shell, Rachael A. Potter, Danielle Griffin, Mark J. Hogan, Shufang Wang, Stefanie Mason, Eddie Darton, Louise Rodino-Klapac   +17 more
doaj   +1 more source

ProteinCoLoc streamlines Bayesian analysis of colocalization in microscopic images

Scientific Reports
Colocalization, the spatial overlap of molecular entities, is often key to support their involvement in common functions. Existing colocalization tools, however, face limitations, particularly because of their basic statistical analysis and their low ...
Manuel Seefelder, Stefan Kochanek, Fabrice A. C. Klein   +2 more
doaj   +1 more source

Adeno‐associated virus serotype 1‐based gene therapy for FTD caused by GRN mutations

Annals of Clinical and Translational Neurology, 2020
Objective Dominant loss‐of‐function mutations in the gene encoding the lysosomal protein, progranulin, cause 5‐10% of frontotemporal dementia cases. As progranulin undergoes secretion and endocytosis, a small number of progranulin‐expressing cells can ...
Christian Hinderer, Rod Miller, Cecilia Dyer, Julia Johansson, Peter Bell, Elizabeth Buza, James M. Wilson   +6 more
doaj   +1 more source

Intron retention is regulated by altered MeCP2-mediated splicing factor recruitment

Nature Communications, 2017
Intron retention is a conserved mechanism that controls gene expression but its regulation is poorly understood. Here, the authors provide evidence that DNA methylation regulates intron retention and find reduced MeCP2 occupancy and splicing factor ...
Justin J. -L. Wong, Dadi Gao, Trung V. Nguyen, Chau-To Kwok, Michelle van Geldermalsen, Rob Middleton, Natalia Pinello, Annora Thoeng, Rajini Nagarajah, Jeff Holst, William Ritchie, John E. J. Rasko   +11 more
doaj   +1 more source

The antiproliferative ELF2 isoform, ELF2B, induces apoptosis in vitro and perturbs early lymphocytic development in vivo

Journal of Hematology & Oncology, 2017
Background ELF2 (E74-like factor 2) also known as NERF (new Ets-related factor), a member of the Ets family of transcription factors, regulates genes important in B and T cell development, cell cycle progression, and angiogenesis. Conserved ELF2 isoforms,
Fiona H. X. Guan, Charles G. Bailey, Cynthia Metierre, Patrick O’Young, Dadi Gao, Teh Liane Khoo, Jeff Holst, John E. J. Rasko   +7 more
doaj   +1 more source

AAV gene replacement therapy for treating MPS IIIC: Facilitating bystander effects via EV‐mRNA cargo

Journal of Extracellular Vesicles
MPS IIIC is a lysosomal storage disease caused by mutations in heparan‐α‐glucosaminide N‐acetyltransferase (HGSNAT), for which no treatment is available.
Tierra A. Bobo, Michael Robinson, Christopher Tofade, Marina Sokolski‐Papkov, Peter Nichols, Stephen Vorobiov, Haiyan Fu   +6 more
doaj   +1 more source

Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy [PDF]

, 2018
Dongsheng Duan
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Human Cytomegalovirus Immediate Early 86-kDa Protein Blocks Transcription and Induces Degradation of the Immature Interleukin-1β Protein during Virion-Mediated Activation of the AIM2 Inflammasome

mBio, 2019
Secretion of interleukin-1β (IL-1β) represents a fundamental innate immune response to microbial infection that, at the molecular level, occurs following activation of proteolytic caspases that cleave the immature protein into a secretable form.
Sara Botto, Jinu Abraham, Nobuyo Mizuno, Kara Pryke, Bryan Gall, Igor Landais, Daniel N. Streblow, Klaus J. Fruh, Victor R. DeFilippis   +8 more
doaj   +1 more source

Combined therapy with oncolytic adenoviruses encoding TRAIL and IL-12 genes markedly suppressed human hepatocellular carcinoma both in vitro and in an orthotopic transplanted mouse model [PDF]

, 2016
Adel Galal El-Shemi, Ahmad Mohammed Ashshi, Youjin Na, Yan Li, Mohammad A. BaSalamah, Faisal A. Al-Allaf, Eonju Oh, Bo‐Kyeong Jung, Chae‐Ok Yun   +8 more
openalex   +1 more source