Therapeutics News and Research

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Metabolic driver of Parkinson’s offers new target for treatment

Researchers have identified a key enzyme driving forms of Parkinson’s, and have shown how blocking it restores normal function in animal and cell models – offering a promising new drug target for the condition.

24 Apr 2025

Bacterial toxin may contribute to rise in early-onset colorectal cancer

In an effort to explain a modern medical mystery, an international team of researchers led by the University of California San Diego has identified a potential microbial culprit behind the alarming rise in early-onset colorectal cancer: a bacterial toxin called colibactin.

23 Apr 2025

Scientists uncover surprising new link between gut health and blood cancer risk

Scientists at Cincinnati Children's along with an international team of researchers have discovered a surprising new connection between gut health and blood cancer risk-one that could transform how we think about aging, inflammation, and the early stages of leukemia.

23 Apr 2025

New approach targets cell surface protein to treat leukemia and solid tumors

In 2021, research led by Ryan Flynn, MD, PhD, and his mentor, Nobel laureate Carolyn Bertozzi, PhD, opened a new chapter in biology, characterizing a new kind of player on the cell surface: glycoRNAs.

23 Apr 2025

DT-109 shows promise in treating atherosclerosis and liver disease

A drug candidate, previously successful at treating severe fatty liver disease, reduces atherosclerosis - a primary driver of cardiovascular death worldwide - in large mammals, a study suggests.

23 Apr 2025

Interferon regulatory factor 8 identified as key driver of liver fat buildup in NAFLD

Non-alcoholic fatty liver disease (NAFLD) is a hepatic metabolic syndrome resulting from lipid metabolic imbalance. Interferon regulatory factor 8 (IRF8) is well known for its roles in immune cell differentiation and tumor pathogenesis; however, its role in regulating glucose homeostasis and lipid metabolism remains unclear.

22 Apr 2025

Machine learning unlocks millions of safer CRISPR enzymes for gene editing

Genome editing has advanced at a rapid pace with promising results for treating genetic conditions-but there is always room for improvement.

22 Apr 2025

Cancer immunotherapy effective across all ages despite immune differences

Older adults with cancer respond just as well as younger patients to immune checkpoint inhibitors despite age-related immune system differences, according to a study by researchers from the Johns Hopkins Kimmel Cancer Center and its Bloomberg~Kimmel Institute for Cancer Immunotherapy, and the Johns Hopkins Convergence Institute.

21 Apr 2025

New therapeutic frontiers in the management of achondroplasia

A recent publication in Genes & Diseases has delivered a compelling synthesis of the latest insights into the cellular mechanisms and therapeutic interventions for achondroplasia, the most common form of genetic dwarfism.

20 Apr 2025

Molecular drivers and emerging treatments in anaplastic thyroid cancer

Anaplastic thyroid cancer (ATC), a rare yet highly aggressive malignancy, continues to represent a major clinical challenge.

20 Apr 2025

GOT2 as a metabolic and immunologic target in pancreatic cancer

A new wave of scientific interest is spotlighting GOT2—glutamic-oxaloacetic transaminase 2—as a compelling therapeutic target in the fight against pancreatic cancer, one of the most lethal and treatment-resistant malignancies.

20 Apr 2025

Reversing adipose tissue fibrosis through targeted microRNA therapies

A new review published in Genes & Diseases highlights the transformative role of microRNAs (miRNAs) in regulating and potentially reversing adipose tissue fibrosis, a condition closely linked to obesity, diabetes, and cardiovascular disease.

20 Apr 2025

Exploring citrullination as a key regulator of gene expression and disease

Citrullination, a transformative protein post-translational modification, is gaining recognition for its wide-ranging impact on cellular function and human disease.

20 Apr 2025

Lipid metabolism as a driver of therapy resistance in triple negative breast cancer

Lipid metabolism has emerged as a central player in the progression and therapy resistance of breast cancer, particularly the aggressive subtype known as triple-negative breast cancer (TNBC).

20 Apr 2025

Redefining cardiovascular medicine through the lens of sncRNAs

In a transformative review, small noncoding RNAs (sncRNAs) have emerged as pivotal regulators in the complex landscape of cardiovascular diseases.

20 Apr 2025

Unmasking Nrf2 as a key driver of treatment resistance in osteosarcoma

A deeper understanding of osteosarcoma, the most common primary malignant bone tumor affecting children and adolescents, is reshaping strategies for overcoming treatment resistance.

20 Apr 2025

Exploring the impact of R-loops on genome integrity and disease

This new review article highlights the pivotal and paradoxical role of R-loops in maintaining genomic stability while simultaneously posing risks to it.

19 Apr 2025

Targeting PGM3 offers new hope against deadly brain tumor

Researchers have found that targeting an enzyme called PGM3 can help stop the growth of glioblastoma, the most dangerous type of brain tumor.

18 Apr 2025

Common pinworm treatment may halt and reverse cancer growth in Merkel cell carcinoma

A common pinworm medication may stop and reverse cancer growth in Merkel cell carcinoma, an aggressive form of skin cancer, according to research led by University of Arizona Cancer Center researchers and published in the Journal of Clinical Investigation.

18 Apr 2025

Some lung cancer patients maintain long-term control after stopping immunotherapy

A subset of patients with non-small cell lung cancer (NSCLC) who discontinued immune checkpoint inhibitor (ICI) therapy due to immune-related adverse events (irAEs) continued to experience long-term disease control.

18 Apr 2025