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New CRISPR breakthrough could transform sickle cell treatment
Researchers at UNSW Sydney have unveiled a next-generation CRISPR tool that could lead to safer treatments for genetic disorders, including Sickle Cell.
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Epigenetics
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ecDNA discovery targets ‘zombie’ cells in childhood cancers
Researchers have discovered how circular extra-chromosomal DNA drives relapse in childhood cancers and found a way to target dormant “zombie” tumour cells – which could allow for the development of more durable treatments.
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New drug combo targets PRMT5 to fight deadly cancers
Scientists at Virginia Tech’s Fralin Biomedical Research Institute have identified a promising drug target – PRMT5 – that could make treatment-resistant lung, brain and pancreatic cancers more vulnerable to therapy.
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QTX153 reverses Rett symptoms and crosses key drug delivery barrier
QTX153, a novel HDAC6 inhibitor, has shown significant symptom reversal in preclinical models of Rett syndrome. This represents progress toward a therapy for a condition with no approved options.
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Disabling the SETD1B enzyme halts leukaemia cell growth
Japanese researchers have identified the epigenetic enzyme SETD1B as a key driver of aggressive acute myeloid leukaemia (AML) – which could lead to new treatment strategies targeting the cancer’s underlying biology in the future.
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‘Delete-to-recruit’: new gene-editing method targets sickle cell
Researchers have developed a novel gene therapy approach that reactivates dormant genes by repositioning them closer to genetic switches called enhancers - showing promise for treating blood disorders like sickle cell disease.
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Google’s AI co-scientist accelerates drug development
Google has introduced an AI-powered 'co-scientist' designed to accelerate biomedical research and drug discovery by generating scientific hypotheses and identifying novel therapeutic targets.
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Study reveals how neurotransmitters influence brain gene expression
A new study demonstrates how neurotransmitters affect brain gene expression, offering potential therapeutic insights for circadian rhythm disorders.
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Differentiated therapies: Unlocking stem cell medicine with epigenetics
The human body has an incredible ability to repair itself, both from acute trauma and chronic damage that accumulates as we age. Here, Cameron Lee, Principal Scientist at Tune Therapeutics, reveals the potential of harnessing the body’s natural regenerative capabilities with epigenetic control of stem cells.
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Creating retroelement-based epigenetic clocks
Targeting the epigenetic states of specific retroelements in the human genome could mitigate the biological effects of ageing.
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Restoring TERT to attenuate ageing hallmarks
In this Q&A, Dr Ronald DePinho of MD Anderson elucidates their preclinical proof-of-concept that adjusting TERT levels could be a viable therapeutic approach for mitigating age-related diseases, such as cancer, heart disease and Alzheimer’s.
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Studying the epigenetic profile of beta cells in insulinoma
Researchers found that tumour beta cells lose their repression marks and express elevated levels of oncogenes.
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Uncovering the genetic and cellular impacts of smoking
Epigenetic data assessing the effects of smoking on DNA methylation could serve as biomarkers of exposure effects.
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How 3D human tissue models are transforming drug discovery
In this Q&A, Keith Murphy, CEO of Organovo, discusses how the shift from animal models to 3D human tissues can transform the preclinical space. Among his many insights, he details the limitations of animal models, how Organovo’s 3D human tissue models facilitate a deeper understanding of genetic expression within specific…
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Unexpected patterns in DNA mutations identified
Disruptions in TP53 and RB1 are key influencers that cause changes in the risk of mutations across chromosomes.
