Results 71 to 80 of about 1,003,750 (315)

Long-term reestablishment of alpha-L-iduronidase activity in MPS I fibroblasts after non-viral gene transfer

open access: yesClinical and Biomedical Research, 2017
Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disorder due to deficiency of alpha-L-iduronidase (IDUA). Limitations such as need of weekly injection, high morbidity and mortality  and high cost of the current treatments show the need for ...
Fabiano de Oliveira Poswar   +5 more
doaj  

Response to combination therapy with interferon alfa-2a and ribavirin in chronic hepatitis C according to a TNF-alpha promoter polymorphism [PDF]

open access: yes, 2003
Background. Tumor necrosis factor-alpha (TNF-alpha) is involved in the pathogenesis of chronic active hepatitis C. Polymorphisms in the promoter region of the TNF-alpha gene can alter the TNF-alpha expression and modify the host immune response.
Konig, A.   +8 more
core   +1 more source

Experience With Performing Rheocarna Therapy via the Single‐Needle Method for Treatment of Chronic Limb‐Threatening Ischemia

open access: yesTherapeutic Apheresis and Dialysis, EarlyView.
ABSTRACT Introduction This study investigated the safety and efficacy of single‐needle Rheocarna therapy for chronic limb‐threatening ischemia (CLTI) with wounds. Methods Six patients with CLTI involving ulcers unresponsive to revascularization underwent single‐needle Rheocarna treatment.
Yasutaka Yamauchi   +9 more
wiley   +1 more source

Enteropathogenic E. coli shows delayed attachment and host response in human jejunum organoid‐derived monolayers compared to HeLa cells

open access: yesFEBS Letters, EarlyView.
Enteropathogenic E. coli (EPEC) infects the human intestinal epithelium, resulting in severe illness and diarrhoea. In this study, we compared the infection of cancer‐derived cell lines with human organoid‐derived models of the small intestine. We observed a delayed in attachment, inflammation and cell death on primary cells, indicating that host ...
Mastura Neyazi   +5 more
wiley   +1 more source

Clinical Practice Recommendations on Genetic Testing of CYP2C9 and VKORC1 Variants in Warfarin Therapy [PDF]

open access: yes, 2015
OBJECTIVE To systematically review evidence on genetic variants influencing outcomes during warfarin therapy and provide practice recommendations addressing the key questions: (1) Should genetic testing be performed in patients with an indication for ...
Shaw, Kaitlyn   +9 more
core   +1 more source

Color Doppler Score: A New Approach for Monitoring a Large Placental Chorioangioma

open access: yesCase Reports in Obstetrics and Gynecology, 2014
We employed color Doppler score as an innovative approach for the prenatal diagnosis and monitoring of a large placental chorioangioma case diagnosed at 26 weeks and the subjective semiquantitative assessment of the vascularization.
Maria Angelica Zoppi   +7 more
doaj   +1 more source

Antisense Oligonucleotide (AON)-based Therapy for Leber Congenital Amaurosis Caused by a Frequent Mutation in CEP290

open access: yesMolecular Therapy: Nucleic Acids, 2012
Leber congenital amaurosis (LCA) is the most severe form of inherited retinal degeneration, with an onset in the first year of life. The most frequent mutation that causes LCA, present in at least 10% of individuals with LCA from North-American and ...
Rob WJ Collin   +5 more
doaj   +1 more source

Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases

open access: yes
Neurological disease due to single gene defects represents a targetable entity for adeno-associated virus (AAV) mediated gene therapy. The delivery of AAV-mediated gene therapy to the brain is challenging, owing to the presence of the blood-brain barrier.
Vardar, Zeynep   +6 more
core   +1 more source

Organoids in pediatric cancer research

open access: yesFEBS Letters, EarlyView.
Organoid technology has revolutionized cancer research, yet its application in pediatric oncology remains limited. Recent advances have enabled the development of pediatric tumor organoids, offering new insights into disease biology, treatment response, and interactions with the tumor microenvironment.
Carla Ríos Arceo, Jarno Drost
wiley   +1 more source

Splicing modulation therapy in the treatment of genetic diseases

open access: yes, 2014
Virginia Arechavala-Gomeza,1 Bernard Khoo,2 Annemieke Aartsma-Rus3 1Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Bizkaia, Spain; 2Endocrinology, Division of Medicine, University College London, London, UK; 3Department ...
Khoo B   +2 more
core  

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