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 successfully tested gene therapy combined with low-dose chemotherapy in five patients aged 7-24 with worsening immune systems due to SCID-X1. The therapy showed substantial improvements in immunity and clinical status, particularly in the first two patients who received treatment.
A new gene therapy approach has successfully reduced the number of epileptic seizures in test animals, mimicking human temporal lobe epilepsy. The treatment involves delivering genes for neuropeptide Y and its receptors into the brain, resulting in a significant reduction in seizures for up to 80% of the animals.
A genetic study of 35,000 patients with inflammatory bowel disease (IBD) reveals new insights into disease progression and severity. The research suggests that IBD may be an array of bowel disorders, rather than a single condition, and could lead to more effective treatments if identified early.
Researchers at Stanford University School of Medicine have identified a gene that appears to reduce the risk of heart failure and improve treatment outcomes. The gene codes for a protein involved in regulating sleep, appetite, and blood pressure, which may play a protective role in the heart.
Researchers have made encouraging results using gene therapy to treat cystic fibrosis by inserting a healthy copy of the CFTR gene into affected cells. The approach restored chloride and fluid transport in both mouse models and human-derived cell cultures, suggesting a potential cure for the genetic disorder.
Researchers have identified the Jagged1 gene as a potential target for therapies to combat Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration. Increasing expression of JAG1 was shown to prevent the development of degenerative disease in zebrafish models.
Researchers have developed a gene therapy approach that delays symptoms and extends lifespan in dogs with a comparable disease to Batten disease. The treatment involves delivering a working version of a gene to produce a key enzyme, resulting in striking clinical improvements and slowed disease progression.
A study published in JAMA Oncology found that superlatives like 'breakthrough' and 'miracle' were used to describe unapproved cancer drugs, often without clinical data. Targeted therapy was the most commonly referenced class of drugs, with immunologic checkpoint inhibitors also being frequently described as 'game-changers'.
Biotech expert Mitchell Finer shares his experiences and strategies for achieving commercial success, including the importance of clinical data and access to capital. He also discusses the growth of companies like bluebird bio and Cell Genesys, and the potential for gene therapy to revolutionize treatment options.
Researchers at King's College London have identified a new gene, HIFalpha, that regulates nerve signals from damaged mitochondria in fruit flies. Switching off the HIFalpha gene restores nerve function and prevents early failure of nerve cells caused by mitochondrial damage.
A phase III clinical trial's results may lead to the first approved gene therapy product in the US for treating Leber congenital amaurosis type 2 (LCA2). The treatment, SPK-RPE65, could improve patients' ability to see and function at night or in dimmer light levels.
Researchers have developed a new mouse model for spinal muscular atrophy that responds to therapy, allowing for post-symptomatic treatment and potentially improving outcomes for patients. The treatment, an antisense oligonucleotide, restores motor unit function in the muscles even after symptoms have begun.
Researchers analyzed genetic material from over 500 CLL patients, identifying dozens of genetic abnormalities linked to the disease. The study found that specific mutations affect treatment response and disease progression, providing insights into the complex interplay between different mutations in cancer cells.
A study funded by the NIH has shown that gene therapy can preserve vision in dogs with late-stage retinitis pigmentosa. The treatment halted disease-associated cell death and preserved photoreceptor structure, improving visual performance under dim light conditions. Further research is needed to assess its safety and potential toxicity...
A Penn study shows that gene therapy can cure canine X-linked retinitis pigmentosa, even when treatment is started at mid- or late-stage disease. The treatment successfully halted photoreceptor cell loss and maintained vision in dogs for over three years.
A new RNA editing technique called exon skipping has shown promising results in treating a rare and severe form of muscular dystrophy. The treatment, licensed to the Kurt+Peter Foundation, aims to slow down muscle decline and stabilize function in individuals with Limb Girdle Muscular Dystrophy Type 2C.
A University of Copenhagen study reveals that an immune gene, Interferon-beta, may prevent Parkinson's disease and dementia. IFNβ-gene therapy successfully prevented neuronal death in experimental models.
Kazan researchers compared direct gene and blood cell-mediated therapy for spinal cord injuries, revealing similar recovery outcomes in rats. The study found that both therapies preserved myelinated fibers, with cell-based therapy extending therapeutic influence over longer distances.
Researchers develop novel mouse model for Leber hereditary optic neuropathy and demonstrate successful gene therapy in improving visual function. The approach could aid therapy development for various mitochondrial diseases, with a clinical trial currently underway.
Bamosiran reduced IOP by similar magnitude across four doses, but failed to demonstrate non-inferiority to timolol in total population; showed excellent tolerability and non-inferiority at dose 1.125% in patients with baseline IOP >=25mmHg
Researchers characterized two genetic mutations linked to achromatopsia in dogs and found that they destabilized molecular channels essential to light signal transduction. The findings provide new insights into the molecular cause of blindness and present opportunities for conducting preclinical assessments of curative gene therapy.
Researchers have identified new mutations in six genes that may explain why some patients respond to a class of anti-cancer drugs known as EGFR inhibitors. These discoveries were made using human tumors grafted into mice, called tumorgraft avatars, which provide a unique environment for studying individual tumor responses to therapy.
Researchers have found five genetic regions that play major roles in cystic fibrosis disease severity, which could lead to new therapies. The study analyzed over 6,000 patients and identified genetic variations associated with lung disease.
A new gene therapy approach delivers the ITGA7 gene to mice with Duchenne muscular dystrophy, reducing symptoms and extending life span by over 10 weeks. The method overcomes previous immunological issues, making it a promising novel treatment for DMD.
Researchers found a promising target for new therapies in genes affected by praziquantel treatment, including a proton pump inhibited by omeprazole. Combining sub-lethal doses of praziquantel with omeprazole killed more parasites than praziquantel alone.
Researchers have developed a novel software that translates the effects of cancer mutations on protein function, revealing how disease mutations target and damage protein signaling networks within human cells. The discovery has significant implications for personalized precision cancer treatments.
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...
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.
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.
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 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.
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.
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.
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.
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.
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 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.