The availability of effective antiretroviral therapy has lowered the risk, and the severity, of neural sequelae of HIV infection. “Early in the HIV pandemic, approximately 15% of people with HIV had dementia and or encephalitis,” Howard Fox told his audience. “Fortunately, with treatment, the prevalence of these severe disorders has been greatly lowered. But there is persistence of what are called more minor disorders – which are not minor if you have them.”
Qingdao Putaike Biomedical Technology Co. Ltd. has described androgen receptor full length (AR-FL) and/or androgen receptor variant 7 (AR-v7) degradation inducers reported to be useful for the treatment of acne vulgaris, androgenic alopecia, cancer, hirsutism, metabolic diseases, polycystic ovary syndrome, precocious puberty and benign prostatic hyperplasia, among others.
Shenzhen Zhongge Biotechnology Co. Ltd. has identified proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase binding moiety covalently linked to an interleukin-1 receptor-associated kinase 4 (IRAK-4) targeting moiety via a linker reported to be useful for the treatment of cancer, autoimmune disorders, inflammatory disorders, transplant rejection, thromboembolism, atherosclerosis, myocardial infarction and metabolic syndrome.
Xi’an Xintong Medicine Research Co. Ltd. has disclosed thyroid hormone receptor (THR)-β agonists reported to be useful for the treatment of atherosclerosis, hypercholesterolemia, hyperlipidemia, hepatic steatosis, thyroid cancer, diabetes, obesity and hypothyroidism.
Researchers from Life Edit Therapeutics Inc. recently reported preclinical data on the application of their gene editing technology Life Edit CRISPR system to Huntington’s disease (HD).
Cystic fibrosis (CF) is a life-threatening autosomal recessive disease affecting over 160,000 people worldwide. CF is caused by loss-of-function mutations in the CF transmembrane conductance regulator (CFTR) that mediates Cl and HCO3 anion transport.
To identify new genetic modifiers for epidermolysis bullosa simplex (EBS), a team led by scientists at Tel Aviv Medical Center performed exome sequencing of 195 patients with EBS from 90 different families, followed by screening for pathogenic variants in selected individuals, which resulted in identification of 3 variants in HMCN1 (codes for hemicentin-1) that co-segregated with the disease phenotype severity in 4 families.
Silicosis, resulting from prolonged exposure to free crystalline silica dust, is an occupational disease characterized by silica nodules and extensive lung tissue fibrosis. The etiology of silicosis remains unclear, challenging early detection and effective treatment.
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