Results 71 to 80 of about 172,174 (293)
Lentiviral manipulation of gene expression in human adult and embryonic stem cells [PDF]
, 2006 Human stem cells could revolutionize the field of medicine by providing a diverse range of cell types for tissue replacement therapies and drug discovery.
Joanne Crossley +11 more
core +1 more source
Recent Advances in the Treatment of Hemophilia: A Review
Biologics: Targets & Therapy, 2021 Emanuela Marchesini,1 Massimo Morfini,2 Leonard Valentino3,4 1Hemophilia Centre, SC Vascular and Emergency Department, University of Perugia, Perugia, Italy; 2Italian Association of Haemophilia Centres (AICE), Naples, Italy; 3National Hemophilia ...
Marchesini E, Morfini M, Valentino L
doaj
COMP–PMEPA1 axis promotes epithelial‐to‐mesenchymal transition in breast cancer cells
Molecular Oncology, EarlyView.This study reveals that cartilage oligomeric matrix protein (COMP) promotes epithelial‐to‐mesenchymal transition (EMT) in breast cancer. We identify PMEPA1 (protein TMEPAI) as a novel COMP‐binding partner that mediates EMT via binding to the TSP domains of COMP, establishing the COMP–PMEPA1 axis as a key EMT driver in breast cancer.
Konstantinos S. Papadakos +6 more
wiley +1 more source
Targeted approaches to induce immune tolerance for Pompe disease therapy
Molecular Therapy: Methods & Clinical Development, 2016 Enzyme and gene replacement strategies have developed into viable therapeutic approaches for the treatment of Pompe disease (acid α-glucosidase (GAA) deficiency).
Phillip A Doerfler +5 more
doaj +1 more source
Molecular Oncology, EarlyView.Glioma cells mainly express the endothelin receptor EDNRB, while EDNRA is restricted to a perivascular tumor subpopulation. Endothelin signaling reduces glioma cell proliferation while promoting migration and a proneural‐to‐mesenchymal transition associated with poor prognosis. This pathway activates Ca2+, K+, ERK, and STAT3 signalings and is regulated
Donovan Pineau +36 more
wiley +1 more source
Autoimmunity, Recessive Diseases, and Gene Replacement Therapy
Molecular Therapy, 2010 The next chapter in the complicated story of host immune responses to in vivo gene therapy has been written in the form of a report on adeno-associated vector (AAV)-mediated gene transfer to muscle of subjects with Duchenne's muscular dystrophy (DMD).1 In this phase I study, six boys with DMD received an intramuscular injection of an AAV vector ...
openaire +2 more sources
Molecular Oncology, EarlyView.Cytarabine is a key therapy for acute myeloid leukaemia (AML), but its efficacy is limited by the dNTPase SAMHD1, which hydrolyses its active metabolite. Screening nucleotide biosynthesis inhibitors revealed that IMPDH inhibitors selectively sensitise SAMHD1‐proficient AML cells to cytarabine.
Miriam Yagüe‐Capilla +9 more
wiley +1 more source
Molecular Oncology, EarlyView.Keratin 19 (KRT19) is overexpressed in high‐grade serous ovarian cancer with high levels of Kallikrein‐related peptidases (KLK) 4–7 and is associated with poor survival. In vivo analyses demonstrate that elevated KRT19 increases peritoneal tumour burden.
Sophia Bielesch +13 more
wiley +1 more source
Lentiviral vectors for gene therapy of Gaucher disease
, 2015 Gaucher disease (GD), a recessive disorder characterised by hepatosplenomegaly, pancytopenia and skeletal complications, is caused by deficiency of the enzyme glucocerebrosidase (GC).
Aitchison, KL
core
Molecular Oncology, EarlyView.We have established a humanized orthotopic patient‐derived xenograft (Hu‐oPDX) mouse model of high‐grade serous ovarian cancer (HGSOC) that recapitulates human tumor–immune interactions. Using combined anti‐PD‐L1/anti‐CD73 immunotherapy, we demonstrate the model's improved biological relevance and enhanced translational value for preclinical ...
Luka Tandaric +10 more
wiley +1 more source