Articles tagged with Gene Therapy
Researchers at Harvard Medical School and Massachusetts General Hospital developed two new strategies to reduce off-target effects of CRISPR/Cas9 genome editing. These techniques use truncated guide RNA molecules and the addition of a FokI domain to the Cas9 protein, resulting in highly specific genome editing.
The CRISPR/Cas system has revolutionized biological research, enabling targeted DNA changes in living cells. This innovation is now being applied to clinical medicine, promising significant advancements in drug therapies, agriculture, and food products.
Researchers at OHSU develop breakthrough technique to replace diseased tissue in patients with mitochondrial disease, paving the way for regenerative medicine treatments. The study uses mitochondrial replacement to create healthy mitochondria from patient skin cells, opening doors to a world of cure options.
Researchers found that vision-restoring gene therapy leads to strengthening of visual pathways in the brain, even after decades of near blindness. The treatment restored nearly intact retina-brain connections, suggesting that unused pathways rebuilt themselves.
A new study demonstrates that gene therapy can give life-long protection to photoreceptor cells in a mouse model of retinitis pigmentosa. The preserved cells were able to drive visually-guided behaviour even in later stages of the condition and despite becoming less sensitive to light.
Researchers have developed a novel control system to regulate therapeutic transgene expression by targeting the passenger strand of a specific microRNA. This approach achieves safe and specific regulation while sparing endogenous gene expression, offering potential for new gene therapy applications.
Researchers at Boston Children's Hospital and Harvard Medical School used gene therapy to restore hearing in mice with genetic deafness. The treatment successfully restored the ability of sensory hair cells to respond to sound and partially restored actual hearing in deaf mice.
A phase 2 trial has shown that gene therapy can improve lung function in people with cystic fibrosis, with a significant benefit seen in patients who received the treatment. The technique uses inhaled molecules of DNA to deliver a normal working copy of the CFTR gene to lung cells.
Researchers found that microglia infiltrate the retina and create a cup-like structure over photoreceptors, accelerating their death. Inhibiting phagocytosis or targeting microglial activation may help preserve vision in retinitis pigmentosa.
A UK trial has shown significant benefits in lung function for patients with cystic fibrosis who received gene therapy. The treatment involves replacing the faulty gene responsible for the disease and was found to be safe and effective. Further research is needed to improve its effectiveness before it can be used as a clinical treatment.
A clinical trial is underway at the Royal Alexandra Hospital to test a new gene therapy treatment for choroideremia, a genetic disorder that causes inherited blindness. The treatment involves injecting a customized virus into the retina to transfer a healthy gene, with promising results already seen in early patients.
Researchers at UNC School of Medicine have developed an experimental gene transfer-based treatment to deliver a missing gene into the cerebrospinal fluid of children with GAN. The treatment approach uses a genetically modified virus to deliver the gigaxonin gene, which is expected to pave the way for treatments for other related diseases.
Researchers at PolyU have developed a novel big data analysis platform that unveils the unregulated patterns of gene network in cancer. The platform discovers potential diagnostic and therapeutic target genes, including Nucleophosmin (NPM1), for Chronic Myelogenous Leukemia (CML).
Australian researchers discovered that a single genetic change in Smchd1 affects its function in the cell, leading to debilitating muscle wasting in FSHD. This fundamental understanding could help develop future treatments for the currently untreatable disease.
Researchers at the University of Pittsburgh School of Medicine have developed a gene therapy that reduces production of brain protein α-synuclein, preventing Parkinson's disease progression in an animal study. The therapy targets mitochondria and α-synuclein interaction, showing promising results in protecting dopamine neurons.
A collaboration between Queensland University of Technology and Avesthagen will translate research findings into a diagnostic and therapeutic tool for migraine. The targeted vitamin therapy has shown reduced migraine disability, frequency, and severity in clinical trials.
Researchers have identified a serotonin receptor, HTR7, as a key mediator of eczema and other forms of itch. The study suggests that targeting this receptor may lead to the development of powerful new therapies for chronic itch conditions.
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 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 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 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.
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.
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.
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.
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 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.
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.
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 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.