Gene News and Research

Latest Gene News and Research

Texas Children’s successfully treats AADC deficiency using FDA-approved gene therapy

Texas Children's is pleased to announce that a three-year-old girl has been successfully treated with the first-ever FDA-approved gene therapy treatment for AADC deficiency.

19 Aug 2025

Improving gene therapy safety with human kidney organoids

Ryuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, "AAV for gene therapy drives a nephrotoxic response via NFκB in kidney organoids."

19 Aug 2025

Why zinc may be the missing nutrient in childhood asthma care

The role of zinc in childhood asthma is critical; deficiency may worsen symptoms, while supplementation could provide a safe, effective treatment alternative.

19 Aug 2025

New strategies to tackle resistant foodborne pathogens

Innovative approaches in food and beverage analysis are transforming pathogen detection and control, emphasizing the role of natural compounds and sequencing.

19 Aug 2025

Targeting a sugar metabolism pathway offers hope for treating rare and aggressive childhood cancer

A new study delving into the genetic drivers of a rare and aggressive childhood cancer called Malignant Peripheral Nerve Sheath Tumor (MPNST) has revealed metabolic frailties in the cancer cells that might be exploited to improved treatments for patients.

18 Aug 2025

Special robot hunts for the perfect cancer warriors

Cancer patients can regain full health with immunotherapy. Now researchers are hunting for the perfect immune cell with the help of a very special robot.

18 Aug 2025

Fucosylation emerges as a key player in digestive inflammatory diseases and cancers

Fucosylation, a crucial post-translational modification, has emerged as a significant factor influencing digestive inflammatory diseases and cancers.

18 Aug 2025

Dual vector gene therapy for GM2 gangliosidosis shows encouraging results in clinical trial

Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a biochemical correction of the disease with minimal adverse reactions.

18 Aug 2025

Vaccines trigger immediate changes in lymph node tissue landscape

Vaccines trigger a notably rapid response in the stromal cells of draining lymph nodes within the first hours after administration.

18 Aug 2025

Polβ enzyme found to protect developing brain from harmful DNA mutations

A research group led by The University of Osaka has discovered that the DNA repair enzyme Polβ plays a crucial role in protecting the developing brain from harmful mutations.

18 Aug 2025

Inactivation of stress pathway allows ER+ breast cancer cells to evade treatment

A team of scientists at the Garvan Institute of Medical Research has discovered that inactivation of a stress pathway makes ER+ breast cancer cells ignore stress signals, allowing them to evade treatment.

18 Aug 2025

Advancing cancer treatment with targeted microrobotic swarms

Cancer remains one of the most challenging diseases to treat, as conventional therapies like chemotherapy and radiotherapy are often hindered by poor specificity, systemic toxicity, and damage to healthy tissues.

18 Aug 2025

Targeting mitochondrial biogenesis to improve outcomes in acute kidney injury

Acute kidney injury (AKI) remains a significant global health challenge, with high mortality rates and the potential for progression to chronic kidney disease.

18 Aug 2025

Exploring the complex origins of endothelial cells in tumor angiogenesis

This newly published review article offers a comprehensive examination of the complexities of tumor angiogenesis and the origins of endothelial cells (ECs) within tumors.

18 Aug 2025

Missing messenger RNA fragments unlock new immunotherapy targets in pediatric brain tumors

A new study, led by researchers at Children's Hospital of Philadelphia (CHOP), identified tiny pieces of messenger RNA that are missing in pediatric high-grade glioma tumors but not in normal brain tissues.

18 Aug 2025

Lactylation drives tumor progression and immune evasion in triple negative breast cancer

Breast cancer maintains its position as the most prevalent malignancy in women worldwide, with triple-negative breast cancer (TNBC) representing the most treatment-resistant subtype due to limited therapeutic targets and frequent relapse.

18 Aug 2025

New AAV gene therapy reverses age-related macular degeneration

Neovascular age-related macular degeneration (nAMD) is a progressive eye disease characterized by choroidal neovascularization and subretinal hemorrhage and exudation, leading to vision impairment. Current first-line treatment for nAMD are anti-VEGF agents, which has shown promising therapeutics outcomes.

18 Aug 2025

RUNX1C isoform identified as driver of chemoresistance in acute myeloid leukemia

One of the biggest challenges in cancer treatment is that certain cancers reappear after chemotherapy-and an aggressive type of blood cancer called acute myeloid leukemia (AML) is notorious for this.

17 Aug 2025

Alzheimer’s gene risks differ by ancestry, major study reveals 133 new variants

Researchers used whole-genome sequencing from diverse populations to identify 156 variants, including 133 novel ones, linked to Alzheimer’s disease and related dementias. The study highlights ancestry-specific genetic risk and protective factors, challenging one-size-fits-all approaches to dementia genetics.

17 Aug 2025

Study shows probiotics help preterm babies fight antibiotic-resistant bacteria

Researchers found that probiotics help reduce antibiotic-resistant bacteria in the guts of very-low-birth-weight preterm infants, but cannot fully prevent resistance genes from spreading. Antibiotic use still promotes gene swapping, highlighting the limits of probiotics alone in neonatal care.

17 Aug 2025

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Gene News and Research

Latest Gene News and Research

Texas Children’s successfully treats AADC deficiency using FDA-approved gene therapy

Improving gene therapy safety with human kidney organoids

Why zinc may be the missing nutrient in childhood asthma care

New strategies to tackle resistant foodborne pathogens

Targeting a sugar metabolism pathway offers hope for treating rare and aggressive childhood cancer

Special robot hunts for the perfect cancer warriors

Fucosylation emerges as a key player in digestive inflammatory diseases and cancers

Dual vector gene therapy for GM2 gangliosidosis shows encouraging results in clinical trial

Vaccines trigger immediate changes in lymph node tissue landscape

Polβ enzyme found to protect developing brain from harmful DNA mutations

Inactivation of stress pathway allows ER+ breast cancer cells to evade treatment

Advancing cancer treatment with targeted microrobotic swarms

Targeting mitochondrial biogenesis to improve outcomes in acute kidney injury

Exploring the complex origins of endothelial cells in tumor angiogenesis

Missing messenger RNA fragments unlock new immunotherapy targets in pediatric brain tumors

Lactylation drives tumor progression and immune evasion in triple negative breast cancer

New AAV gene therapy reverses age-related macular degeneration

RUNX1C isoform identified as driver of chemoresistance in acute myeloid leukemia

Alzheimer’s gene risks differ by ancestry, major study reveals 133 new variants

Study shows probiotics help preterm babies fight antibiotic-resistant bacteria

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