Articles tagged with Gene Therapy
Jin Kim Montclare's new research aims to deliver genes and drugs simultaneously, enabling the potential treatment of multi-drug resistant cancer cells. The engineered protein-lipid system breaks down naturally in the body, offering a safer alternative to traditional polymers.
A new enzyme-replacement therapy, Sebelipase Alfa, has been shown to effectively treat Lysosomal Acid Lipase Deficiency by reducing disease-related liver and blood cholesterol abnormalities. The therapy, which targets hepatocytes, resulted in lower cholesterol levels and reduced liver fat content in patients with the condition.
The new 2015 WHO Classification of Lung Tumors provides standard criteria for pathology diagnosis and clinical practice. The updated classification focuses on histologic subtyping and classification according to predominant subtype, leading to multiple new discoveries in lung adenocarcinoma.
Scientists have successfully used gene therapy to fully restore vision in a mouse model of Leber congenital amaurosis-1, a genetic disorder causing severe visual impairment. The treatment, which replaced the deficient retGC1 protein, showed long-lasting results and supports clinical testing for human patients.
A review article highlights barriers to overcome in CAR T cell therapy for lymphoma, including physical barriers and immunosuppression. The study explores factors related to tumor biology and immunology compared to treatment response in patients with lymphoma.
A recent study published in SLEEP reveals that African genetic ancestry is associated with lower slow-wave sleep amounts in African-American adults. The research found a significant genetic basis for racial differences in slow-wave sleep, which may lead to the development of population-specific treatment approaches.
Researchers at Oregon State University have made a significant advance in photodynamic therapy to combat ovarian cancer, achieving complete cancer cell elimination with no regrowth of tumors. The new approach combines existing techniques with compounds that make cancer cells vulnerable to reactive oxygen species, reducing natural defen...
Harvard researchers have reconstructed an ancient virus effective at delivering gene therapies to liver, muscle, and retina. The discovery may lead to novel vectors for safer and more potent gene therapies.
Scientists have developed a new technology that significantly improves the ability to target specific faulty genes and edit them with healthy DNA. The method reduces off-target DNA binding, allowing researchers to produce gene editing tools that are 100 times more specific for their target sequence.
A new test developed by UBC researchers allows physicians to measure the effects of gene silencing therapy in Huntington's disease. The test detects small amounts of toxic protein and can be used to follow changes in brain levels over time.
Researchers reconstructed an ancient virus to deliver gene therapies, targeting liver, muscle, and retina. The discovery could lead to safer and more potent treatments for various diseases.
Researchers have recreated the evolutionary lineage of AAVs to reconstruct an ancient viral particle that is highly effective at delivering gene therapies. The approach could lead to designing a new class of genetic drugs that are safer and more potent than those currently available.
A new study suggests that exogenous glucagon-like peptide 2 (GLP-2) treatment can improve the excretion of toxic bile acids and stimulate liver growth in neonatal pigs with parenteral nutrition-associated liver disease. This finding supports a beneficial role for GLP-2 as a novel therapy in PNALD.
A modified version of a protein that suppresses female reproductive organ development has been shown to inhibit the growth of chemotherapy-resistant ovarian tumors in animal models. Researchers at Massachusetts General Hospital have developed a gene therapy approach using a viral vector to deliver this protein, which resulted in signif...
Scientists have decoded the genome and transcriptome of an incurable subtype of acute lymphoblastic leukemia, revealing a novel program associated with leukemic cells. The study identifies key genes that trigger a reprogramming of the leukemia cells, leading to promising drug tests, including Venetoclax.
Researchers successfully deliver an RNA encoding alpha-1-antitrypsin (AAT) into cells, enabling them to produce highly functional AAT. This approach offers a safe and cost-effective solution for treating single gene disorders like AAT deficiency.
Scientists found that additional genes can compensate for knocked-out genes and mitigate consequences. In a study on zebrafish, researchers identified emilin 3B as a rescuing gene for the egfl7 gene, which regulates blood vessel growth.
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 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 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 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.
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 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.
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 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.