Results 51 to 60 of about 7,531,202 (384)
Clinical 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 +2 more sources
Challenges in constructing genetic instruments for pharmacologic therapies [PDF]
arXiv, 2020 The genes that encode the targets of most therapies do not have rare variants with large-effect or common variants with moderate effects on the biomarker reflecting the pharmacologic action of the corresponding therapy. Therefore, providing genetic target validation for most therapies is challenging.
arxiv
Standing genetic variation and the evolution of drug resistance in HIV [PDF]
, 2011 Drug resistance remains a major problem for the treatment of HIV. Resistance can occur due to mutations that were present before treatment starts or due to mutations that occur during treatment. The relative importance of these two sources is unknown.
arxiv +1 more source
FoxO1 signaling in B cell malignancies and its therapeutic targeting
FEBS Letters, EarlyView.FoxO1 has context‐specific tumor suppressor or oncogenic character in myeloid and B cell malignancies. This includes tumor‐promoting properties such as stemness maintenance and DNA damage tolerance in acute leukemias, or regulation of cell proliferation and survival, or migration in mature B cell malignancies.
Krystof Hlavac +3 more
wiley +1 more source
GMP-manufactured CRISPR/Cas9 technology as an advantageous tool to support cancer immunotherapy
Journal of Experimental & Clinical Cancer ResearchBackground CRISPR/Cas9 system to treat human-related diseases has achieved significant results and, even if its potential application in cancer research is improving, the application of this approach in clinical practice is still a nascent technology ...
M Caforio +4 more
doaj +1 more source
Regulatory Framework for Advanced Therapy Medicinal Products in Europe and United States
Frontiers in Pharmacology, 2019 Advanced therapy medicinal products (ATMPs) are a fast-growing field of innovative therapies. The European Union (EU) and the United States (US) are fostering their development.
Carolina Iglesias-Lopez +5 more
doaj +1 more source
Messenger RNA therapy for rare genetic metabolic diseases
Gut, 2019 Decades of intense research in molecular biology and biochemistry are fructifying in the emergence of therapeutic messenger RNAs (mRNA) as a new class of drugs.
P. Berraondo +3 more
semanticscholar +1 more source
FEBS Letters, EarlyView.Metabolic dysfunction‐associated steatotic liver disease (MASLD) affects nearly one‐third of the global population and poses a significant risk of progression to cirrhosis or liver cancer. Here, we discuss the roles of hepatic dendritic cell subtypes in MASLD, highlighting their distinct contributions to disease initiation and progression, and their ...
Camilla Klaimi +3 more
wiley +1 more source
Male Infertility: Genetics, Mechanism, and Therapies [PDF]
BioMed Research International, 2016 The World Health Organization declaimed that infertility is a major global public health issue of the last few decades. Infertility is commonly defined as the failure to conceive after 1 year of unprotected intercourse and is estimated to concern 72.4 million people worldwide with 40.5 million currently seeking medical care.
Coutton, Charles +4 more
openaire +4 more sources
Insights into PI3K/AKT signaling in B cell development and chronic lymphocytic leukemia
FEBS Letters, EarlyView.This Review explores how the phosphoinositide 3‐kinase and protein kinase B pathway shapes B cell development and drives chronic lymphocytic leukemia, a common blood cancer. It examines how signaling levels affect disease progression, addresses treatment challenges, and introduces novel experimental strategies to improve therapies and patient outcomes.
Maike Buchner
wiley +1 more source