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Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
Researchers are exploring the potential use of HDAC inhibitors, a group of drugs known for their capability to impede tumour cell division, to reduce the pain and damage associated with sickle cell disease. The drugs have shown initial evidence of reactivating the gene responsible for producing foetal haemoglobin, which cannot…
USC researcher Dr Steven Gazal and his team have identified human genome base pairs that have remained constant over millions of years of mammalian evolution. These base pairs are linked to human disease. Using DNA from 240 mammal species, they identified genetic variations affecting an individual's survival and reproduction.
A recent study from researchers at the University of California examines the BRCA2 gene, which plays a crucial role in repairing damaged DNA, and possible links with increased risk of breast and ovarian cancer.
US researchers suggest that combing a dual gene-editing approach with antiretroviral drugs can eliminate HIV infections in animal models.
Researchers from the Netherlands have utilised organoids and the CRISPR-Cas9 "molecular scissor" system to better understand the features and biology of fibrolamellar carcinoma (FLC), a rare type of liver cancer that affects adolescents and young adults.
Australian researchers uncover a gene that controls inflammation in kidney disease, which could pave the way for more precise disease diagnostics and personalised treatments.
US researchers find combination of chloroquine and venetoclax promotes cancer cell death in mouse models with acute myeloid leukaemia (AML).
St. Jude Children's Research Hospital, US, scientists created a new combination therapy method to tackle drug resistance in a type of leukaemia with KMT2A gene rearrangement.
Using gene therapy, the researchers corrected abnormal heart rhythms by restoring a protein that heart cells need to establish connections with one another.
Lipid nanoparticles have been used to encapsulate CRISPR-Cas9 and deliver it to cells in mice, where it was highly effective at knocking down expression of a target protein.
US researchers evaluate an experimental gene therapy in the first ever nonhuman primate model for Usher Syndrome.
US researchers spotlight how p53, the most frequently mutated tumour suppressor gene, can be activated against cancer cells.
The scientists say that the engineered CRISPR enzymes could overcome key limitations for eventual use to treat genetic diseases irrespective of a patient’s particular mutation.
The researchers developed nanoparticles able to penetrate the neural retina and deliver mRNA to the photoreceptor cells whose proper function makes vision possible.
Using lung basal cell organoids, researchers identify a gene that directs the development of lung cancer and offers a better understanding of its disease treatment.
