Results 331 to 340 of about 12,497,887 (407)

Polymeric nanoparticle-mediated GBA1 gene therapy is neuroprotective in a preclinical model of Parkinson's disease. [PDF]

Drug Deliv Transl Res
Kwatra M, Kwak G, Li H, Suk JS, Ko HS.
europepmc   +1 more source

Transcriptional regulation of neuropeptide receptors underlies context‐dependent adaptation in Drosophila melanogaster

FEBS Open Bio, EarlyView.
Under environmental changes, the expression level of neuropeptide (NP) and neuropeptide receptor (NPR) genes changes to confer context‐dependent adaptation to the model organism Drosophila melanogaster. Through finding more regulatory elements in the NPR genes in comparison with their ligands (NPs), we found that NPR‐biased transcriptional regulation ...
SeungHeui Ryu, Yanan Wei, Zekun Wu, Tianmu Zhang, DoHoon Lee, Hadi Najafi, Woo Jae Kim   +6 more
wiley   +1 more source

Hematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia. [PDF]

Mol Ther Methods Clin Dev
Luthers CR, Mittelhauser A, Colamartino A, Wu X, Cirigliano S, Long JD, Sanchez JM, Romero Z, Kohn DB.   +8 more
europepmc   +1 more source

A unified model for Duchenne muscular dystrophy gene involvement in cancer: context‐dependent tumour suppression and oncogenicity

FEBS Open Bio, EarlyView.
We propose a context‐dependent model where the Duchenne muscular dystrophy (DMD) gene acts as a tumour suppressor in aggressive tumours and as an oncogene in less aggressive ones. We propose this model as a unified framework to explain the opposing survival associations with DMD expression and to guide experimental exploration of the dual role of DMD ...
Lee Machado, Leanne Jones, Sonika Divakar, Michael Naidoo, Karen Anthony   +4 more
wiley   +1 more source

Long-term data needed for SCD gene therapy. [PDF]

Blood Adv
Leonard A.
europepmc   +1 more source

Report on the 2nd MObility for Vesicle research in Europe (MOVE) symposium—2024

FEBS Open Bio, EarlyView.
The 2nd MObility for Vesicle research in Europe (MOVE) Symposium in Belgrade brought over 280 attendees from 28 countries to advance extracellular vesicle (EV) research. Featuring keynotes, presentations, and industry sessions, it covered EV biogenesis, biomarkers, therapies, and manufacturing.
Dorival Mendes Rodrigues‐Junior, Michaela Kocholata, Marilena E. Lekka, Olga Janouskova, Moran Yadid, Maja Kosanovic   +5 more
wiley   +1 more source

Retinal gene therapy using epiretinal AAV-containing fibrin hydrogel implants. [PDF]

Sci Adv
Scruggs BA, Berger A, Knudsen T, Kopp FN, Hill M, Trncic E, Anderson K, Iezzi R, Marmorstein AD.   +8 more
europepmc   +1 more source

Identification of amino acids essential for angulin‐1/3 binding of the tricellular tight junction binder, angubindin‐1

FEBS Open Bio, EarlyView.
Angubindin‐1 binds angulin‐1/‐3 at tricellular tight junctions, enhancing intestinal macromolecule permeation. Alanine scanning identified six essential residues (L562, L598, E638, V640, Y643, and K644) of angubindin‐1 critical for binding to angulin‐1/‐3 and permeation‐enhancing activity, providing insights for the development of targeted noninvasive ...
Taiki Kuzu, Yumi Iwashita, Keisuke Tachibana, Itsuki Nishino, Yuki Niwa, Atsuko Uyeda, Kazuki Matsuo, Masahiro Nagahama, Masuo Kondoh   +8 more
wiley   +1 more source

Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B. [PDF]

N Engl J Med
Reiss UM, Davidoff AM, Tuddenham EGD, Chowdary P, McIntosh J, Muczynski V, Journou M, Simini G, Ireland L, Mohamed S, Riddell A, Pie AJ, Hall A, Quaglia A, Mangles S, Mahlangu J, Haley K, Recht M, Shen YM, Halka KG, Fortner G, Morton CL, Gu Z, Hayden RT, Neufeld EJ, Okhomina VI, Kang G, Nathwani AC.   +27 more
europepmc   +1 more source