Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
Researchers discover that different shapes of alpha-synuclein aggregates cause distinct neurodegenerative diseases, including Parkinson's disease and Multiple System Atrophy. The study suggests new possibilities for treating brain diseases with a drug that counteracts aggregate development.
Researchers have identified a gene, PRDM8, linked to early loss of night vision in animal models. Gene therapy for Leber's congenital amaurosis shows promising results, but temporary improvements are seen.
Researchers have developed a novel delivery strategy using an aptamer to introduce functional microRNAs into cells, targeting breast cancer and blood vessel cells. The therapy shows anti-cancer and angiogenic activities, inhibiting tumor growth and protecting against atherosclerosis.
Dr. Qizhen Shi's team aims to investigate a novel gene therapy approach that induces immune tolerance for hemophilia A, overcoming the challenge of inhibitory antibodies. The project seeks to develop a clinically translatable gene therapy protocol that provides therapeutic FVIII protein.
A Phase III clinical trial has shown that combined lumacaftor-ivacaftor therapy improves lung function and lowers pulmonary exacerbation rates in cystic fibrosis patients with the F508del genetic mutation. The treatment is expected to benefit around 15,000 US patients.
Dr. Ana Buj Bello, a leading expert in gene therapy, has received the Outstanding New Investigator Award for her groundbreaking research on treating myotubular myopathy through gene therapy. Her work has shown promising results in animal models and paves the way for a potential clinical trial to treat children with this rare disease.
Researchers have found a molecular 'switch' that safely controls severe side effects associated with haploidentical stem cell transplantation. The switch, inducible caspase 9 (iC9), is activated by a single dose of bio-inert chemical and clears symptoms without jeopardizing graft function.
A new study by geneticists from the University of Leicester has identified nearly 80 genes associated with 'morningness' and 'eveningness' in fruit flies. This discovery could lead to better diagnostics and personalized medicine, where individuals receive tailored therapies based on their unique genetic profiles.
The TGen study identified over 50 rare disorders associated with muscle contractures and stiff joints in newborns. The researchers matched these conditions to specific genes on the X chromosome, offering potential therapies for diagnosis and treatment.
Researchers developed a unique approach using microscopic gas bubbles to deliver directly to cancer cells a viral gene therapy in combination with an experimental drug. The treatment strategy used a novel 'cancer terminator virus' and demonstrated enhanced prostate cancer cell death while sparing healthy cells in preclinical experiments.
Researchers successfully restored bilirubin levels to those found in healthy animals using gene therapy, offering new hope for the treatment of Crigler-Najjar syndrome. The therapy involved transferring a copy of the UGT1A1 gene into liver cells using an AAV vector, resulting in long-term correction without immunosuppression.
Researchers at the University of Manchester have developed a new genetic profiling approach to identify patients with right lung cancer who are eligible for targeted therapies. The method uses smaller tissue biopsies and has been shown to detect genetic changes in over 95% of samples.
Gene therapy for Leber congenital amaurosis improves vision and retina sensitivity within weeks of treatment, with peak benefits after one to three years. However, these gains diminish over six years, revealing the need for further research to sustain restored vision.
A new study demonstrates that gene therapy for Leber congenital amaurosis (LCA) improves vision in patients within weeks of treatment, with benefits peaking at one to three years after treatment. However, the visual gain eventually declines, highlighting the need for future therapeutic strategies to sustain restored vision.
Researchers have developed a new therapy using naïve cells to prevent cytomegalovirus, Epstein-Barr virus, and adenovirus infections in cord blood transplant patients. The treatment has shown safety and efficacy in clinical trials.
Researchers at Icahn School of Medicine at Mount Sinai have developed a gene therapy approach that targets and corrects genetic mutations linked to heart failure. The treatment uses molecular scissors to cut out diseased genes and replace them with normal ones, restoring healthy cardiac cell function.
A new therapeutic strategy has been identified for treating colorectal cancer with the BRAF mutated gene, which is associated with poor prognosis. Inhibition of endosomal activity and P45-IKKα enzyme inhibition show promise in preventing tumour growth and metastasis.
Researchers identified a genetic pathway linked to poor response to corticosteroid treatment in children with asthma. Targeting the VNN-1 pathway may offer new therapeutic strategies for difficult-to-treat patients.
A study published in JAMA found that gene therapy resulted in clinical improvement for children with Wiskott-Aldrich syndrome, a rare immunodeficiency disorder. The treatment, which involved infusing corrected stem cells back into patients, led to the resolution of severe eczema and recurring infections.
A new gene therapy treatment has shown significant clinical improvement in six patients with Wiskott-Aldrich Syndrome, a rare and complex immune disorder. The treatment involves correcting genetic anomalies in blood stem cells using a lentiviral vector, resulting in restored immune function and reduced symptoms.
A new scoring method called MATH measures genetic variability among cancer cells within tumors from patients with head and neck cancer. High MATH scores correspond to tumors with many differences among gene mutations present in different cancer cells, correlating with lower patient survival.
A study suggests that sequencing tumor genomes without comparing them to a patient's noncancerous tissue can lead to inaccurate treatment decisions and side effects. The researchers analyzed over 800 cancer patients' data, finding that nearly half of the identified genetic changes were not related to the cancer.
A recent study published in Science Translational Medicine found that many genetic alterations identified using tumor-only sequencing are not associated with cancer, but rather reflect inherited germline mutations. The study analyzed DNA from 815 patients and found that nearly half of the detected genetic alterations were 'false positi...
A new study comparing gene therapy to half-matched transplants for SCID-X1 reveals that gene therapy leads to faster immune development, fewer infections, and reduced hospitalizations. Gene therapy shows promise as a viable alternative treatment for infants with this rare immune disorder.
Researchers at University of British Columbia developed a new technology called Hi-Fi SELEX to accelerate and improve aptamer selection. The platform enhances diversity and enables high-fidelity amplification of therapeutically useful reagents.
Researchers at UAB have created an artificial virus that can self-assemble, surround DNA fragments, and reach the nucleus of cells without biological risk. This innovation provides a promising alternative to traditional viruses in gene therapy, enabling the efficient delivery of therapeutic DNA and drugs directly to target cells.
Duke researchers have developed a new method to precisely control gene activity by chemically manipulating proteins that package DNA. This technology allows for the activation of specific gene promoters and enhancers, which could provide a new avenue for gene therapies and guiding stem cell differentiation.
Researchers mapped genetic changes in tumor samples from 10 men with prostate cancer, revealing a common ancestral cell and shared gene faults. The study provides insight into how prostate cancer spreads and evolves, potentially leading to new treatment targets and personalized approaches.
Researchers at ETH Zurich have developed a novel biotechnological solution to treat erectile dysfunction by triggering reliable erections with gene therapy and blue light exposure. The system works similarly across all mammals, including humans, and is expected to have few side effects.
A potential treatment for myotonic muscular dystrophy has been identified, using an experimental drug that improves muscle function and reduces symptoms in mice. The therapy targets excessive activity of a cellular protein called TWEAK and its receptor Fn14, which correlates with disease severity.
Researchers have successfully treated dogs with hemophilia using a mutant human protein with unusually high blood-clotting power. The treatment eliminated pre-existing antibodies and reversed the bleeding disorder in two years without causing thrombosis or immune reactions.
Researchers at the University of Birmingham have developed a method to genetically engineer immune cells to combat Nasopharyngeal carcinoma (NPC), a third most common cancer in Chinese males. The engineered T-cells use Epstein-Barr virus presence to fight NPC, offering a promising new treatment approach.
Scientists have developed a new mechanism for engineering traits governed by multiple genes, paving the way for personalized gene therapies and regenerative medicine. The approach uses the Cas9 protein to activate specific genes, allowing for precise control over multiple genes and potentially treating diseases.
Research suggests statin therapy provides greatest benefit to patients with highest genetic risk of heart attack. Genetic analysis assesses heart attack risk independently of traditional factors, identifying those at higher baseline risk who reap greater benefits from therapy. The study's findings have implications for precision medici...
Researchers identified a genetic risk score that predicts cardiovascular death and heart attack risk, with individuals in the highest genetic risk group experiencing a significant reduction in cardiovascular risk with statin therapy. The study found a three-fold increase in absolute risk reduction in those with high genetic risk.
Researchers at Boston University School of Medicine identified a specific genetic signal, miR-10b-5p, that strongly correlates with disease severity and extent of neuronal death in Huntington's disease. This discovery may provide a faster and more effective way to determine the effectiveness of treatments.
Researchers have devised a clonal strategy to ensure adult epidermal stem cells meet the highest safety criteria before use in treatments. The approach involves cultivating, genetically modifying and testing individual cells to guarantee their viability and safety.
Researchers at Indiana University identified a unique gene signature in pancreatic cancer patients who can benefit from targeted therapies cutting off the growth pathways fed by abnormal blood vessels. This finding suggests that these patients may benefit from precision medicine approaches.
Researchers at UT Dallas have created a novel gene-delivery system that shuts down after delivering a gene, offering a potential new strategy for treating diseases. The approach sidesteps health problems associated with permanent gene alteration.
Pioneers in gene transfer technology, Drs. Mulligan and Miller developed the first DNA-based vectors to stably transfect cells with selectable markers. Their work laid the foundation for retroviral vector-based gene therapy strategies, leading to clinical testing and potential treatment of genetic diseases.
Researchers at Johns Hopkins Medicine have developed a new nanoparticle-based gene therapy that effectively kills brain cancer cells in rats and lengthens their survival. The treatment uses biodegradable nanoparticles filled with genes for an enzyme that turns a compound into a potent killer of cancer cells.
A recent study reveals that sea slugs can absorb genes from the algae they eat, enabling them to photosynthesize and survive for months on sunlight. This natural process has implications for gene therapy and rapid evolution in multicellular species.
Researchers at the University of Maryland have discovered a new mechanism of inheritance in roundworms that could revolutionize our understanding of evolution and disease treatment. The team found that genes can be silenced through a process called RNA interference, and this silencing can persist for multiple generations.
A study found that multiplexed genetic screening for EGFR and ALK gene rearrangements followed by molecularly-guided treatment is cost-effective compared to standard chemotherapy. The approach offers better outcomes in terms of quality-adjusted life years gained and life-years, making it a valuable option for patients with NSCLC.
Researchers at Henry Ford Hospital identified a gene that may contribute to keloid scar development. The study found altered AHNAK gene expression in keloid tissue, suggesting a potential target for novel treatments.
Researchers have identified a critical safety parameter related to AAV gene therapy, which can increase liver cancer risk. They found that modifying the vector to avoid targeting specific genes reduces cancer incidence, paving the way for safer gene therapies.
Scientists have identified several genetic mutations in the Ebola virus that could render sequence-based treatments ineffective. The study found 10 new mutations that may interfere with monoclonal antibody, siRNA, and PMO drugs, highlighting the need to consider genetic drift when developing potential therapeutics.
A study found that AAV vector integration and microRNA expression influenced liver cancer development after gene therapy. The study highlights the importance of considering vector design features when designing AAV vectors for gene therapy.
An international research team identified a critical gene linked to childhood blindness and vision loss. The study reveals that mutations in this gene affect phospholipid metabolism, leading to photoreceptor death and vision impairment.
Researchers have identified distinct gene pathways altered by infection and diet in vascular tissue, suggesting future therapies may need to be tailored. This discovery sheds light on the complex development of atherosclerosis and its association with infections like Porphyromonas gingivalis and Chlamydia pneumoniae.
Researchers at CNIO have developed a gene therapy that reactivates the telomerase gene only in the heart of adult mice, resulting in increased survival rates and regeneration of cardiomyocytes. This study provides proof-of-concept for treating chronic and acute heart failure and opens new avenues for age-related diseases.
Katherine High, Amit Nathwani, Arthur Nienhuis, and Andrew Davidoff honored for their groundbreaking research on gene therapy for hemophilia B. They developed successful clinical applications using adeno-associated virus vectors and demonstrated stable therapeutic expression of the clotting factor.
James Ingle, a renowned breast cancer expert at Mayo Clinic, has been awarded the 2014 William L. McGuire Memorial Lecture Award for his groundbreaking research on pharmacogenomics in breast cancer treatment. His work aims to develop personalized approaches to prevent breast cancer in high-risk women by identifying genetic biomarkers t...
A new genetic therapy has restored light response to the retinas of blind mice and dogs, offering hope for future clinical trials in humans. The therapy uses a virus to insert a gene into normally blind cells, followed by photoswitches that activate the cells when exposed to light.
Researchers found that a specific protein's conformational defect causes Autosomal Dominant Lateral Temporal Lobe Epilepsy, a form of familial epilepsy. Treatment with chemical chaperone LGI1 mutant protein was shown to ameliorate seizure susceptibility in mice.
Researchers have discovered novel combinations of targeted therapies and chemotherapy regimens that improve survival outcomes for patients with historically poor-prognosis leukemia. These findings, presented at the American Society of Hematology Annual Meeting, offer new hope for elderly and aggressive genetic mutation patients.
Scientists have made a breakthrough in treating epidermolysis bullosa by using induced pluripotent stem cells to grow back healthy skin cells. The new technique, called therapeutic reprogramming, was tested on mice and showed promising results.
Researchers have discovered a gene variant, PHACTR1, that reduces the risk of cervical artery dissection and other related conditions. This finding could lead to new treatment strategies for stroke prevention in young adults.
A UCL-led team of scientists has uncovered the structure of pores found in cell nuclei, revealing how they selectively block certain molecules from entering to protect genetic material and normal cell functions. The discovery could lead to the development of new antiviral drugs and better delivery mechanisms for gene therapy.
Researchers use genetically engineered pig models to study human diseases and identify new drug targets for cancer, cardiovascular diseases, diabetes, neurodegenerative diseases, ophthalmology, and xenotransplantation. These models enable the development of novel cell therapies to restore diseased tissues and organs.