XTalks
APRIL 24, 2023
Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.
Drug Discovery World
JULY 3, 2023
PrecisionLife and the University of Oxford have signed a data access agreement to license the Oxford Endometriosis Gene (OXEGENE) dataset to develop new personalised treatments for endometriosis patients. Endometriosis affects 10% of women globally, but on average it takes over seven years for patients to receive a diagnosis.
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XTalks
APRIL 15, 2021
From a total of 30 single-nucleotide polymorphisms (SNPs) in genes previously identified as being associated with blood pressure, many had a relatively neutral effect on the risk of coronary artery disease. Figure 2: Using drug-target MR to validate sympathetic nervous system genes involved in heart failure.
The Pharma Data
NOVEMBER 2, 2020
ONWARD is investigating Adial’s lead drug candidate, AD04, as a therapeutic agent for the treatment of Alcohol Use Disorder (AUD) in persons with certain target genotypes related to the serotonin transporter and receptor genes. Key ONWARD highlights include: All 25 planned investigative sites are active.
XTalks
MARCH 3, 2021
MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene. Molybdopterins are a class of cofactors found in most molybdenum-containing and all tungsten-containing enzymes. It is characterized by a deficiency in molybdenum cofactor production.
XTalks
NOVEMBER 10, 2020
How many genes need to be scanned in order to determine the presence of a genetic disease? HS: Due to the heterogeneity of the rare disease population, there are many relevant gene lists, and the genomic data analysis of these patients covers almost 2,000 different genetic targets.
Drug Discovery World
MAY 2, 2023
The CRISPR-Cas9 system, awarded the Nobel Prize in Chemistry in 2020 for its applications for gene editing, was initially encountered in the bacterial immune system. These modifications lead to alterations in the function of the proteins expressed by these genes, manifesting in cellular processes dependent on those proteins.
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