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Search results for “Gene therapy”

1,000+ results for "Gene therapy"

Scientists put CRISPR on safer path to patient treatments with new process for evaluating impacts of gene edits that alter rather than “knock out” DNA code

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

Could gene therapy soon curb muscle loss in the elderly?

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

What caused the blood malignancies linked to gene therapy for sickle cell disease?

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
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Gene Therapy for the Treatment of Huntington’s Disease

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

Epilepsy research boosts case for new gene therapy for Dravet syndrome

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.

SourceUniversity of Virginia Health System·DateJan 19, 2022

Researchers show increased survival rate in prostate cancer patients with addition of hormone therapy treatments

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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Davis Instruments Vantage Pro2 Weather Station offers research-grade local weather data for networked stations, campuses, and community observatories.

Gene therapy advance could reverse a common genetic cause of hearing loss

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

Experimental gene therapy reverses sickle cell disease for years

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

CRISPR/Cas9 gene editing boosts effectiveness of ultrasound cancer therapy

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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Apple MacBook Pro 14-inch (M4 Pro) powers local ML workloads, large datasets, and multi-display analysis for field and lab teams.

Spinal muscular atrophy: Charité study confirms efficacy of gene therapy

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

Study encourages cautious approach to CRISPR therapeutics

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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Apple iPhone 17 Pro delivers top performance and advanced cameras for field documentation, data collection, and secure research communications.

Gene therapy boosts Parkinson’s disease drug benefits

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

High-dose radiation thwarts tumor growth in patients with advanced lung cancer

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

A decade after gene therapy, children born with deadly immune disorder remain healthy

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
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Treatment with gene therapy of a new rat model of Morquio A, a disease with severe skeletal dysplasia and cartilage deterioration

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

‘Frameshifting’ therapy for mast cell cancers reduces size, spread

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

Children’s Hospital of Philadelphia researchers develop “dimmer switch” to help control gene 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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NIH-funded study finds gene therapy may restore missing enzyme in rare disease

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

Researchers identify new gene that may increase risk of ALS

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

New gene therapy uses Tylenol to combat genetic diseases

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
Meta Quest 3 512GB

Meta Quest 3 512GB enables immersive mission planning, terrain rehearsal, and interactive STEM demos with high-resolution mixed-reality experiences.

Nintendo® wii may help improve balance in children with cerebral palsy

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

Gene therapy offers a potential cure to children born without immune system

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

Gene therapy restores immune function in children with rare immunodeficiency

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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AmScope B120C-5M Compound Microscope supports teaching labs and QA checks with LED illumination, mechanical stage, and included 5MP camera.

Gene therapy offers potential cure to children born without an immune system

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

Intense light may hold answer to dilemma over heart treatment

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 presents novel gene delivery and electrophysiology platforms at ARVO

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.

SourceNanoscope Technologies·DateMay 5, 2021

Researchers identify potential combination therapy for aggressive lung cancer

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-based diagnostics and gene therapy

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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Nikon Monarch 5 8x42 Binoculars deliver bright, sharp views for wildlife surveys, eclipse chases, and quick star-field scans at dark sites.

Bypassing broken genes

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.

SourcePenn State·JournalMolecular Therapy·DateApr 21, 2021

"Molecular Tomographer" algorithm maps gene expression in space

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
Celestron NexStar 8SE Computerized Telescope

Celestron NexStar 8SE Computerized Telescope combines portable Schmidt-Cassegrain optics with GoTo pointing for outreach nights and field campaigns.

Gene therapy shows promise in treating rare eye disease in mice

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.

SourceeLife·DateApr 13, 2021

Bioengineer wins NIH grant to attack cystic fibrosis

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.

SourceRice University·DateApr 8, 2021

Cellular benefits of gene therapy seen decades after treatment

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
Anker Laptop Power Bank 25,000mAh (Triple 100W USB-C)

Anker Laptop Power Bank 25,000mAh (Triple 100W USB-C) keeps Macs, tablets, and meters powered during extended observing runs and remote surveys.

With gene therapy, scientists develop opioid-free solution for chronic pain

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

ACGT awards grant to advance strategies for treating lung cancer

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.

SourceAlliance for Cancer Gene Therapy·DateMar 1, 2021

A Canadian success story: world-first to treat Fabry disease with gene therapy

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

New technology shows potential to improve potency and durability benefits in gene therapy

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
Sony Alpha a7 IV (Body Only)

Sony Alpha a7 IV (Body Only) delivers reliable low-light performance and rugged build for astrophotography, lab documentation, and field expeditions.

Cre-controlled CRISPR: Conditional gene inactivation just got easier

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

Improved vectors for ocular gene therapy

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 potentially safer, more effective gene therapy vector for blood disorders

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
Sky-Watcher EQ6-R Pro Equatorial Mount

Sky-Watcher EQ6-R Pro Equatorial Mount provides precise tracking capacity for deep-sky imaging rigs during long astrophotography sessions.

First hybrid gene therapy shows early promise in treating long QT syndrome

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.

SourceMayo Clinic·JournalCirculation·DateJan 28, 2021

Genome editing to treat human retinal degeneration

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

Genome editing to treat human retinal degeneration

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

Gene therapy strategy found effective in mouse model of hereditary disease TSC

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