Researchers at ChristianaCare's Gene Editing Institute describe a new process for evaluating the impacts of gene edits that alter rather than completely disabling DNA code. The study validates the safety and efficacy of their novel approach for using CRISPR to improve lung cancer treatments.
SourceChristianaCare Gene Editing Institute·JournalGene Therapy·TypeExperimental study·DateMar 21, 2022
Researchers discovered a crucial RNA strand called CYTOR that helps build muscle mass, and found it decreases with age. Gene therapy stimulated CYTOR production, leading to increased fast-twitch muscle fibers and improved muscle function in humans and mice.
SourceNorwegian University of Science and Technology·JournalScience Translational Medicine·TypeExperimental study·DateFeb 23, 2022
A recent study found that gene therapy delivery vectors were unlikely to cause the blood malignancies reported in trials; however, the exact cause remains a mystery. The study's results suggest that cancer risk in sickle cell disease may be more complex than initially thought, warranting further investigation.
SourceBoston Children's Hospital·JournalJournal of Clinical Investigation·DateFeb 15, 2022
Researchers found that dexamethasone administration after gene therapy increased liver transgene expression in mice. This finding suggests a potential strategy for reducing steroid side effects in future clinical trials.
SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalHuman Gene Therapy·TypeCase study·DateJan 31, 2022
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A recent study led by University at Buffalo researchers found that photobiomodulation reduces inflammation, improves blood flow and heals wounds up to 19 days faster. Light therapy accelerated skin healing from cancer radiation therapy by an average of 49-42 days.
SourceUniversity at Buffalo·JournalPhotonics·DateJan 27, 2022
A novel gene therapy approach using an adeno-associated virus (AAV) has shown promising results in treating Huntington's disease. The therapy targets the mutated huntingtin gene (HTT), providing efficient and precise processing of a primary artificial microRNA, leading to potent pharmacological activity for HTT lowering.
SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalHuman Gene Therapy·TypeCase study·DateJan 25, 2022
Researchers developed a gene therapy called Targeted Augmentation of Nuclear Gene Output (TANGO), which boosts SCN1A protein production in brain cells. The treatment restored normal cell function and reduced seizures in lab mice with Dravet syndrome, offering hope for the first direct treatment of the fundamental cause.
A meta-analysis published in The Lancet Oncology reveals that men with intermediate- and high-risk prostate cancer experience improved overall survival with the addition of hormone therapy to radiotherapy. The study found prolonged adjuvant hormone therapy benefits men, while neoadjuvant hormone therapy did not.
SourceUniversity Hospitals Cleveland Medical Center·JournalThe Lancet Oncology·TypeMeta-analysis·DateJan 18, 2022
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Researchers developed a non-muscle targeted gene therapy that enhances muscle fiber repair and improves muscle function in LGMD 2B patients. The treatment, administered via a single injection, reduces muscle degeneration and restores myofiber size and muscle strength.
SourceChildren's National Hospital·JournalJournal of Clinical Investigation·DateJan 4, 2022
A novel gene delivery technique has successfully restored hearing in mice with a mutated STRC gene, which is the second most common genetic cause of hearing loss. The approach could be used to deliver other large genes and may provide a broad window of opportunity for treatment from babies to adults.
SourceBoston Children's Hospital·JournalScience Advances·DateDec 15, 2021
A new gene therapy called LentiGlobin has been shown to completely eliminate episodes of severe pain caused by sickle cell disease and restore blood cells to their normal shape. The treatment uses a patient's own stem cells and has the potential to give people with this disease their life back.
SourceColumbia University Irving Medical Center·JournalNew England Journal of Medicine·TypeRandomized controlled/clinical trial·DateDec 13, 2021
Researchers have developed a CRISPR/Cas9 gene editing system to enhance the effectiveness of sonodynamic therapy, allowing tumors to be effectively shrunk in a mouse model of liver cancer. The technology reduces antioxidant defense systems, increasing cancer cell death from the treatment.
SourceAmerican Chemical Society·JournalACS Central Science·DateDec 8, 2021
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A Charité study confirms the efficacy of gene therapy in improving muscle function and reducing mortality in children with spinal muscular atrophy. The treatment, onasemnogene abeparvoven, was found to be effective in children under two years of age, offering a promising alternative to existing treatments.
SourceCharité - Universitätsmedizin Berlin·JournalThe Lancet Child & Adolescent Health·TypeObservational study·DateNov 26, 2021
A novel gene therapy has shown sustained expression of clotting factor VIII, leading to a reduction or complete elimination of bleeding events in patients with hemophilia A. The trial demonstrated improved production of coagulation factor VIII over prolonged periods.
SourceChildren's Hospital of Philadelphia·JournalNew England Journal of Medicine·DateNov 17, 2021
Researchers from Genethon and Université de Paris have developed an AAV-based gene therapy that corrects skeletal damage caused by XLH in mouse models. The treatment targets liver hepatocytes to express therapeutic proteins, potentially offering a lifelong solution for this rare disease.
A recent study by Sanford Burnham Prebys and the National Cancer Institute has shown that CRISPR-Cas9 gene editing can favor cells with mutated forms of genes linked to cancer. This highlights the importance of monitoring patients undergoing CRISPR-based gene therapy for cancer-related mutations.
SourceSanford Burnham Prebys·JournalNature Communications·DateNov 11, 2021
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A preclinical study shows that gene therapy can restore dopamine production in the brain, eliminating symptoms of Parkinson's disease. The therapy targets the substantia nigra region, where dopamine-releasing neurons reside, and provides a potential target for disease-modifying therapies.
SourceNorthwestern University·JournalNature·TypeExperimental study·DateNov 3, 2021
A new study finds that high-dose radiation therapy can lengthen progression-free survival for people with advanced lung cancer when systemic therapy has not fully halted tumor growth or spread. Stereotactic body radiation therapy (SBRT) shows promise in treating oligoprogressive, metastatic lung and breast cancer.
SourceAmerican Society for Radiation Oncology·DateOct 25, 2021
Researchers report that 90% of patients who received gene therapy between 2009 and 2012 remain disease-free, with significant immune system differences observed among those treated. The therapy is most effective in younger children, but further work is needed to achieve high levels of gene correction in all patients.
SourceUniversity of California - Los Angeles Health Sciences·JournalBlood·TypeRandomized controlled/clinical trial·DateOct 15, 2021
A new study finds that left-sided breast cancer radiation therapy increases the risk of coronary artery disease in younger survivors, with a significant increase in risk seen at 10.5% for those who received left-sided radiation therapy compared to 5.8% for right-sided.
SourceAmerican College of Cardiology·JournalJACC CardioOncology·DateSep 21, 2021
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Researchers at Universitat Autonoma de Barcelona develop gene therapy that fully corrects severe whole-body alterations in Morquio A disease, a rare condition caused by GALNS enzyme deficiency. The therapy, administered via intravenous viral vector, prevents bone malformations and life-threatening complications.
SourceUniversitat Autonoma de Barcelona·JournalNature Communications·TypeExperimental study·DateSep 10, 2021
Researchers developed a 'frameshifting' therapy that alters mRNA to reduce mast cell tumor size and prevent organ infiltration. The treatment, targeting the c-KIT gene, shows promise in reducing KIT protein expression, signaling, and function.
SourceNorth Carolina State University·JournalMolecular Therapy·TypeExperimental study·DateAug 10, 2021
The Massage Therapy Foundation will host the International Massage Therapy Research Conference (IMTRC) on May 12-13, 2022, in Alexandria, Va. The event aims to bring together global experts to share research findings and discuss current innovations in massage therapy.
Researchers at Children's Hospital of Philadelphia have developed a system that can fine-tune protein expression from gene therapy vectors, addressing the need for controlled dosing. The 'dimmer switch' uses alternative RNA splicing and an orally available small molecule to adjust levels of expression up or down as needed.
SourceChildren's Hospital of Philadelphia·JournalNature·TypeExperimental study·DateJul 28, 2021
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A new study suggests that gene therapy delivered into the brain may be safe and effective in treating AADC deficiency. The therapy resulted in clinical improvement of symptoms, including reduced oculogyric crises, improved movement and motor function, and better sleep patterns.
SourceNIH/National Institute of Neurological Disorders and Stroke·JournalNature Communications·DateJul 16, 2021
A new study reveals that light therapy's benefits come from activating the Period1 gene in a brain region involved in mood regulation. Exposure to light at the end of the dark period has an antidepressant effect on mice.
Researchers identified a new gene, TP73, associated with an increased risk of sporadic ALS. Mutations in this gene have been found to interfere with nerve cell health and lead to abnormal cell differentiation and increased cell death. This discovery provides a potential target for therapy development.
SourceAmerican Academy of Neurology·JournalNeurology·DateJun 16, 2021
Researchers developed a novel gene therapy approach that utilizes acetaminophen to correct disease-causing mutations and make liver cells immune to its toxic effects. This allows treated cells to multiply and reduces the need for liver transplants in treating genetic disorders.
SourceOregon Health & Science University·JournalScience Translational Medicine·DateJun 11, 2021
Researchers have developed a platform that produces bespoke star-shaped polypeptide nanoparticles, effectively delivering range of therapies including gene therapies. The material accelerates bone tissue regeneration with a six-fold increase in new bone formation.
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Research suggests that Nintendo Wii therapy can significantly improve balance in children with cerebral palsy, even when combined with conventional physical therapy. The analysis found medium to large effects of the therapy on functional balance and dynamic balance, respectively.
SourceWiley·JournalDevelopmental Medicine & Child Neurology·DateJun 9, 2021
Researchers have found that a truncated version of the otoferlin gene, including its transmembrane domain, is essential for proper neurotransmitter release and sensory cell maturation. This discovery brings scientists closer to developing gene therapy treatments for severe congenital hearing loss.
SourceOregon State University·JournalMolecular Biology of the Cell·DateMay 19, 2021
Researchers developed a gene therapy that successfully treated 48 out of 50 children with ADA-SCID, a rare life-threatening disease. The treatment involves removing stem cells, delivering a new copy of the ADA gene, and returning the corrected cells to produce healthy immune cells.
SourceUniversity College London·JournalNew England Journal of Medicine·DateMay 11, 2021
Infants and children with ADA-SCID, a life-threatening inherited immunodeficiency disorder, have regained immune system function after receiving gene therapy. The treatment involves inserting a normal copy of the ADA gene into blood-forming stem cells, offering a one-time procedure with long-term benefits.
SourceNIH/National Institute of Allergy and Infectious Diseases·JournalNew England Journal of Medicine·DateMay 11, 2021
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A new form of gene therapy has successfully treated 48 out of 50 children born with a rare and deadly inherited disorder. The treatment, developed by an international team, involves delivering a corrected copy of the ADA gene into stem cells, which are then returned to the child's body to produce healthy immune cells.
SourceUniversity of California - Los Angeles Health Sciences·JournalNew England Journal of Medicine·DateMay 11, 2021
Scientists at the University of Colorado Anschutz Medical Campus discovered that intense light can enhance the expression of the ANGPTL4 gene, which regulates fatty lipids in plasma. This could lead to a new tool for safely blocking the gene and reducing cardiovascular risk.
SourceUniversity of Colorado Anschutz Medical Campus·JournalTrends in Molecular Medicine·DateMay 10, 2021
Nanoscope Technologies is featuring groundbreaking research on optical gene delivery and functional characterization platform technologies for treating retinal diseases. The company's lead product, vMCO-010, delivers light-sensitive Multi-Characteristics Opsin into retinal cells to restore vision in patients with degeneration.
A new study by UCLA researchers identified a novel combination therapy using immune checkpoint inhibitors with ATRA to potentially overcome resistance to immunotherapy in LKB1-deficient lung cancer. The therapy led to eradication of over 70% of tumors and generated durable tumor-specific immunity.
SourceUniversity of California - Los Angeles Health Sciences·JournalCancer Research·DateMay 4, 2021
CRISPR/Cas technology has advanced diagnostics and gene therapy, enabling fast and accurate disease diagnosis and treating untreatable diseases like cancer and blood disorders. Recent advances in CRISPR-Cas technologies are highlighted, with a discussion of the strength and challenges for its future clinical usage.
SourceCompuscript Ltd·JournalBIO Integration·DateApr 26, 2021
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Researchers developed a new approach called Co-opting Regulation Bypass Repair (CRBR) that can correct genetic diseases caused by single gene mutations. The method uses the CRISPR/Cas9 system and non-homologous end joining to insert a functional copy of the gene, enabling treatment in all adult tissues.
The Tomographer algorithm transforms gene-sequencing data into spatially resolved images, mapping gene expression patterns across tissues. By analyzing tissue strips and mRNA measurements, the algorithm reconstructs spatial gene-expression patterns in tissues like the brain of the Australian Bearded Dragon.
SourceEcole Polytechnique Fédérale de Lausanne·JournalNature Biotechnology·DateApr 20, 2021
Researchers at Penn Medicine delivered a new gene therapy that improved vision in three patients with severe vision impairments. The therapy showed sustained improvements in day and night vision, without serious side effects.
SourceUniversity of Pennsylvania School of Medicine·JournaliScience·DateApr 19, 2021
The Alliance for Cancer Gene Therapy Summit 2021 features key findings on translating CAR T-cell therapy success to complex solid tumors. Researchers share insights on evolving cell and gene regulatory processes, innovative science from the lab to the clinic, and tackling challenges in pancreatic cancer and glioblastoma.
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A gene therapy protects eye cells in mice with a rare disorder, suggesting a combination approach may preserve vision in people with retinitis pigmentosa. Researchers found that using Txnip gene therapies, along with treatments for oxidative stress and inflammation, provided additional protection for the cells.
Xue Sherry Gao, a bioengineer at Rice University, has won an NIH grant to develop tools that can correct multiple single mutations causing cystic fibrosis. Her approach uses CRISPR/Cas9-based gene editing to fix the faulty CFTR gene, which is difficult to treat with traditional gene therapy.
A peptide nucleic acid-based gene editing technique has been identified as a crucial part of the gene editing toolkit, offering an alternative to CRISPR-Cas systems. The technique, developed by Carnegie Mellon's Danith Ly and Yale University's Peter Glazer, allows for in vivo gene editing using nanoparticles.
SourceCarnegie Mellon University·JournalNature·DateApr 7, 2021
Scientists at the University of Cambridge have demonstrated a new approach to gene therapy that may help repair damage caused by neurodegenerative conditions like glaucoma and dementia. The study, published in Science Advances, shows promise for treating chronic degenerative diseases.
SourceUniversity of Cambridge·JournalScience Advances·DateMar 31, 2021
A study found that gene therapy can create a long-term store of correct T cells in the human thymus, leading to sustained health in patients with SCID-X1. The treatment works by delivering corrected genes into stem cells, which then produce healthy immune cells.
SourceUniversity College London·JournalNature Communications·DateMar 17, 2021
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Anker Laptop Power Bank 25,000mAh (Triple 100W USB-C) keeps Macs, tablets, and meters powered during extended observing runs and remote surveys.
Researchers at UC San Diego developed a gene therapy that temporarily represses a gene involved in sensing pain, increasing pain tolerance and providing months of relief. The therapy could be used for various chronic pain conditions, including lower back pain and rare neuropathic disorders.
SourceUniversity of California - San Diego·JournalScience Translational Medicine·DateMar 10, 2021
A team led by Dr. Brian Brown is developing a CAR T-cell therapy that specifically targets immune-suppressing macrophages in tumors, clearing the way for other T cells to destroy cancer cells. The grant aims to advance this promising approach and bring new hope to patients with solid tumor cancers.
A Canadian pilot study on gene therapy for Fabry disease shows the treatment is working and safe, enabling patients to produce normal levels of the defective enzyme. The trial, led by Dr. Aneal Khan, treated five men with a single dose of gene therapy, which resulted in stable patients who are no longer requiring replacement therapy.
SourceUniversity Health Network·JournalNature Communications·DateFeb 25, 2021
Researchers have developed an immune tolerance platform called ImmTORTM to overcome challenges in gene therapy, including immunogenicity and durability. The addition of ImmTOR nanoparticles to AAV vectors has shown potential to enhance efficacy, safety, and durability by mediating more efficient transgene expression.
SourceLifeSci Public Relations·JournalJournal of Mathematical Sciences Advances and Applications·DateFeb 24, 2021
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Researchers have developed a new method, Cre-Controlled CRISPR, which combines the benefits of the Cre/lox system and CRISPR/Cas9 genetic scissors for conditional gene inactivation. This approach allows for faster and easier gene editing with reduced labor needed to flank genes with lox sequences.
SourceTechnische Universität Dresden·JournalNature Communications·DateFeb 23, 2021
Researchers have developed improved gene vectors for ocular gene therapy, allowing for widespread delivery and reducing risks associated with traditional approaches. These vectors are being tested in clinical trials and have shown promise in restoring daylight vision in animal models of achromatopsia.
SourceLudwig-Maximilians-Universität München·JournalEMBO Molecular Medicine·DateFeb 22, 2021
A new study published in the Journal of Nuclear Medicine found that combining radionuclide therapy with immunotherapy can slow prostate cancer progression and increase survival time. The treatment promotes prostate cancer immunogenicity, making tumors more receptive to immunotherapy.
SourceSociety of Nuclear Medicine and Molecular Imaging·JournalJournal of Nuclear Medicine·DateFeb 8, 2021
Researchers at Children's Hospital of Philadelphia developed a new gene therapy vector that produces more hemoglobin with a lower dose, minimizing toxic side effects. The vector, ALS20, was found to be significantly more effective than current vectors in treating beta-globinopathies.
SourceChildren's Hospital of Philadelphia·JournalMolecular Therapy·DateJan 29, 2021
Researchers developed a gene therapy strategy to treat Leber congenital amaurosis by adding copies of the normal CRX gene under its native control mechanism. This approach restored some CRX protein function and drove expression of opsins in patient-derived retinal organoids.
SourceNIH/National Eye Institute·JournalStem Cell Reports·DateJan 28, 2021
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Sky-Watcher EQ6-R Pro Equatorial Mount provides precise tracking capacity for deep-sky imaging rigs during long astrophotography sessions.
Researchers at Mayo Clinic have developed the first hybrid gene therapy for treating long QT syndrome, a genetic heart rhythm condition. The therapy targets the KCNQ1 gene and has shown potential therapeutic efficacy in two in vitro model systems using beating heart cells reengineered from patient blood samples.
New gene editing strategies, including CRISPR technologies, are being investigated for treating inherited retinal diseases. Researchers aim to identify the most viable therapeutic approaches using human retinal tissue and organoids.
SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalHuman Gene Therapy·DateJan 25, 2021
New gene editing strategies, including CRISPR technologies, are being investigated for treating inherited retinal diseases. The article highlights the most viable therapeutic approaches and discusses safety concerns and challenges in extending the capabilities of CRISPR-Cas9.
SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalHuman Gene Therapy·DateJan 19, 2021
Researchers at Massachusetts General Hospital have developed a gene therapy strategy that effectively treats mice with a mutated TSC2 gene, causing the growth of noncancerous tumors. The treatment extends survival to 462 days and reduces brain damage in mice, suggesting potential for human clinical trials.
SourceMassachusetts General Hospital·JournalScience Advances·DateJan 8, 2021