Results 51 to 60 of about 1,504,225 (383)
Genetic therapy for the nervous system [PDF]
Human Molecular Genetics, 2011 Genetic therapy is undergoing a renaissance with expansion of viral and synthetic vectors, use of oligonucleotides (RNA and DNA) and sequence-targeted regulatory molecules, as well as genetically modified cells, including induced pluripotent stem cells from the patients themselves. Several clinical trials for neurologic syndromes appear quite promising.
William J. Bowers +2 more
openaire +2 more sources
GENE THERAPY IN MUCOPOLYSACCHARIDOSIS TYPE IIIA: CASE REPORTS
Slovenska pediatrija, 2022 Mucopolysaccharidoses are a group of rare lysosomal storage diseases. The clinical signs develop gradually, the impairment is progressive and multiple organs are affected. With the currently known treatment options, the patient cannot be cured.
Benjamin Lah +7 more
doaj +1 more source
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
Regulating Human Gene Therapy [PDF]
, 1985 Scientific developments have moved the public debate on genetic engineering to the issue of human gene therapy. Because so many important societal values must be weighed in deciding which, if any, of the first protocols for human gene therapy should be ...
Areen, Judith C.
core +1 more source
Targeting Oncogenic BRAF: Past, Present, and Future. [PDF]
, 2019 Identifying recurrent somatic genetic alterations of, and dependency on, the kinase BRAF has enabled a "precision medicine" paradigm to diagnose and treat BRAF-driven tumors.
Bivona, Trever G +2 more
core +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
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
Alcohol addiction: a molecular biology perspective. [PDF]
, 2015 Alcohol misuse represents worldwide an important risk factor for death and disability. Excessive alcohol consumption is widely diffused in different ethnicities and alcohol use is part of the lifestyle of both young and old people.
Ferraguti, Giampiero +2 more
core +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