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Microscopic image of a Wilms tumor of a pediatric kidney

More mutations than previously thought in childhood cancers

May 30, 2025
By Nuala Moran
No Comments
Researchers in the U.K. have overthrown the orthodox view that childhood cancers have a low mutation burden, opening up new drug targets and opportunities for repurposing existing therapies. In particular, a high mutation rate is associated with a response to cancer immunotherapy. But although PD-1 checkpoint inhibitors are approved for treating pediatric cancers with a high level of microsatellite instability mutations, in general it is thought childhood tumors are not amenable to immunotherapy.
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Microscopic image of a Wilms tumor of a pediatric kidney

More mutations than previously thought in childhood cancers

May 29, 2025
By Nuala Moran
No Comments
Researchers in the U.K. have overthrown the orthodox view that childhood cancers have a low mutation burden, opening up new drug targets and opportunities for repurposing existing therapies. In particular, a high mutation rate is associated with a response to cancer immunotherapy. But although PD-1 checkpoint inhibitors are approved for treating pediatric cancers with a high level of microsatellite instability mutations, in general it is thought childhood tumors are not amenable to immunotherapy.
Read More
Liver
Gastrointestinal

Precision medicine approach identifies culprit in alcohol-associated hepatitis

May 29, 2025
By Anette Breindl
No Comments
Researchers have identified KpsM as a virulence factor in Escherichia coli that was responsible for liver damage in alcohol-associated hepatitis (AH). A small-molecule inhibitor of KpsM reduced liver damage in animal models of AH.
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Human aging illustration

Juvenescence's $76M+ to advance CD38, ketone drugs to clinic

May 21, 2025
By Nuala Moran
No Comments
Anti-aging specialist Juvenescence Ltd. reached the first close of its series B-1 at $76 million and said it is on course to close the round at $150 million in the third quarter of 2025. “The reason for the first close and not waiting for the very end is just so we can start to move the pipeline forward,” said Richard Marshall, CEO. “We’ve got molecules in and waiting to go, so the sooner we can get going on those, the better,” he told BioWorld.
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Children’s Hospital of Philadelphia
ASGCT 2025

First bespoke gene editing therapy treats rare metabolic disease

May 20, 2025
By Anette Breindl
No Comments
Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.
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Woman receiving radiotherapy treatments for cancer
Cancer

Amphiregulin drives metastasis through a paradoxical ‘badscopal’ effect of radiotherapy

May 20, 2025
By Coia Dulsat
No Comments
Researchers at the University of Chicago have shed light on the role of tumor-promoting factors induced by radiotherapy and their potential impact on future therapeutic strategies. The article, published in Nature on May 14, 2025, points to radiation-induced amphiregulin as a key driver of tumor metastasis.
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Illustration of kidneys with DNA double helix
Nephrology

ASGCT 2025: Overcoming kidney complexity in gene and cell therapy

May 16, 2025
By Mar de Miguel
No Comments
Gene and cell therapies (GCTs) can target the kidney to treat congenital, acute or chronic diseases affecting this organ. However, its complex structure poses a challenge for these technologies. To be precise and effective in the long term, new approaches should circumvent the specificities of renal tissue, with novel methods of delivery and gene transfer to offer new therapeutic options for patients who lack them.
Read More
Children’s Hospital of Philadelphia
ASGCT 2025

First bespoke gene editing therapy treats rare metabolic disease

May 15, 2025
By Anette Breindl
No Comments
Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.
Read More
Liver illustration
Endocrine/metabolic

Liver is also immune organ, influenced by microbiome

May 14, 2025
By Anette Breindl
No Comments
Immunity is not a function most people particularly associate with the liver. But because of its connection to the gut, the liver is exposed to bacterial metabolites as few other organs are. And when either the liver or the gut is not functioning well, it can adversely affect immunity as well.
Read More
Liver illustration
Endocrine/metabolic

Liver is also immune organ, influenced by microbiome

May 12, 2025
By Anette Breindl
No Comments
Immunity is not a function most people particularly associate with the liver. But because of its connection to the gut, the liver is exposed to bacterial metabolites as few other organs are. And when either the liver or the gut is not functioning well, it can adversely affect immunity as well. The liver is connected to the gut via both the biliary system and the portal vein. Those two conduits allow metabolites from the gut microbiome to influence what’s going on in the liver. Both liver and gut damage can affect this communication for the worse. And surprisingly, one of the consequences is immune dysfunction.
Read More
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