Scientists at the San Raffaele Telethon Institute for Gene Therapy have unlocked a remarkable biological window – a fleeting period immediately after birth when circulating blood stem cells become uniquely accessible for genetic modification directly within the body. This discovery could transform treatment approaches for devastating genetic blood disorders, offering hope without the invasive procedures […]
Researchers at Children’s Hospital of Philadelphia and Penn Medicine have successfully treated an infant with a rare genetic disease using a bespoke CRISPR gene editing therapy, marking a watershed moment in personalised genomic medicine.
Researchers have developed a novel RNA-based gene editing system capable of inserting entire therapeutic genes, addressing limitations of current CRISPR technology for diseases with multiple mutations.
University of Chicago researchers have developed a groundbreaking imaging technology that visualizes the spatial organization of genetic material in three dimensions, without relying on conventional optical instruments. This technique, called volumetric DNA microscopy, works by tagging DNA and RNA molecules and allowing neighboring tags to interact, creating a molecular network that encodes their relative positions […]
Scientists have created the first comprehensive map showing how human DNA is mixed and passed down through generations, offering new understanding of genetic diversity and its relationship to fertility and evolution.
UCLA Health study demonstrates significant impact of genetic diagnostics on treatment decisions and specialist referrals in complex neurodevelopmental cases, pointing towards more personalised medical approaches.
A groundbreaking RNA-based technology allows therapeutic genes that are too large for conventional delivery methods to be successfully transported into cells, offering new hope for treating muscular dystrophies and other genetic disorders caused by mutations in large genes.
New research challenges long-held beliefs about the causes of gout, highlighting the significant impact of genetics on the chronic inflammatory condition.
New research reveals how delaying inflammatory signalling can dramatically increase the number of blood stem cells formed during embryonic development, potentially aiding efforts to produce these cells in the laboratory.
Researchers at The Rockefeller University have identified a rare genetic mutation that increases vulnerability to tuberculosis, potentially reshaping our understanding of tumour necrosis factor’s role in the immune system.
April 2024
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