Gene Therapy News
December 23, 2024
Top Headlines
Dec. 10, 2024 In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...
Nov. 6, 2024 The CRISPR tool is capable of repairing the genetic defect responsible for the immune disease chronic granulomatous disease. However, researchers have now shown that there is a risk of inadvertently introducing other ...
Oct. 28, 2024 Using state-of-the-art technology, researchers have identified several specific steps needed for CRISPR to become active and perform its gene editing function. These preclinical findings could lead ...
Sep. 25, 2024 Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an international Phase III ...
Latest Headlines
updated 10:25pm EST
Sep. 9, 2024 Scientists developed a highly promising gene therapy to treat glaucoma -- a debilitating eye condition that can lead to complete vision loss, and which affects around 80 million people worldwide. The ...
Sep. 9, 2024 Researchers tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that affects the brain, lungs, skin, and skeleton with no ...
Aug. 29, 2024 For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of fixing faulty genes ...
Aug. 20, 2024 A newly discovered code within DNA -- coined 'spatial grammar' -- holds a key to understanding how gene activity is encoded in the human genome. This breakthrough finding revealed a long-postulated ...
Aug. 7, 2024 Bioengineers have developed a noninvasive tool to measure gene expression and gene therapy delivery in specific brain regions using ...
July 24, 2024 Researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with Duchenne ...
July 17, 2024 A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in ...
July 12, 2024 Researchers have used gene editing to restore hearing in adult mice with a type of inherited hearing loss. They showed that shutting down a damaged copy of a gene called a microRNA (miRNA) enabled ...
July 10, 2024 Researchers restored hearing in preclinical mouse models with a specific form of inherited deafness called DFNA50 caused by mutations in microRNA, by ...
June 17, 2024 Researchers have designed a new genetic therapy that could alleviate debilitating giant moles in a rare skin ...
Earlier Headlines
June 13, 2024 A viral gene therapy has reversed some brain abnormalities in infant mice with FOXG1 syndrome, a significant step toward one day treating children with this severe neurodevelopmental ...
June 10, 2024 Scientists have improved a gene-editing technology that is now capable of inserting or substituting entire genes in the genome in human cells efficiently enough to be potentially useful for ...
May 30, 2024 Researchers have developed a novel version of a key CRISPR gene-editing protein that shows efficient editing activity and is small enough to be packaged within a non-pathogenic virus that can deliver ...
May 29, 2024 Bioengineers report that a foaming liquid worked better than a standard liquid formulation at transferring gene therapy components to cells in laboratory ...
Apr. 30, 2024 In an effort to improve delivery of costly medical treatments, a team of researchers in electrical engineering has developed a stimulating method that could make the human body more receptive to ...
Apr. 24, 2024 In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range ...
Apr. 22, 2024 Clarifying the cause of a skin disease led to the discovery of a new disease-causing gene, a new category of diseases, and new perspectives for both counseling and therapy. The discovery is the first ...
Apr. 16, 2024 A recent breakthrough study has shown potential to improve therapeutic outcomes for patients suffering from lung ...
Apr. 10, 2024 New research sheds light on the significance of the glucocorticoid receptor in drug-resistant prostate cancer, showing that the development of drug resistance could be prevented by limiting the ...
Apr. 4, 2024 Researchers have succeeded in correcting a gene defect that causes a hereditary liver disease and its adverse effects on ...
Mar. 21, 2024 Researchers have discovered that a specific mutation in the cancer cells of an aggressive type of blood cancer can prevent novel immunotherapies such as CAR T-cell therapy from working. Their study ...
Mar. 6, 2024 A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, muscular ...
Mar. 5, 2024 Researchers who work with tiny drug carriers known as lipid nanoparticles have developed a new type of material capable of reaching the lungs and the eyes, an important step toward genetic therapy ...
Mar. 4, 2024 In a discovery that opens the door to a less invasive way of treating some serious disorders before birth, UC San Francisco scientists have found that delivering medicine through amniotic fluid is as ...
Feb. 23, 2024 A new study identified a set of 140 genes that may help predict enhanced disease-free survival in patients with non-small cell lung cancer (NSCLC) treated with a combination of immunotherapy and ...
Feb. 2, 2024 A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead ...
Jan. 17, 2024 Axi-cel CAR T targets the CD19 molecule on large B-cell lymphoma cells. The ZUMA-7 trial demonstrated that axi-cel reduced the risk of disease progression, the need for new therapy, or death by 60% ...
Jan. 2, 2024 Just like a doctor adjusts the dose of a medication to the patient's needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also ...
Dec. 13, 2023 A previously mysterious small RNA molecule in mice is found to play a crucial role in gene expression, and may be the first identified member of a new class of regulatory ...
Dec. 7, 2023 Researchers have laid the foundation for the development of a gene therapy for the genetic heart disease arrhythmogenic cardiomyopathy (ACM). Their approach, based on replacement of the PKP2 gene, ...
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- New Gene Editing Tool Helps Zero in on Small Cancer-Linked Mutations
- Gene Therapy Hope for Children With Kidney Disease
- Scientists Reverse Hearing Loss in Mice
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- Calcium: Important Not Just for Your Bones but Also for Your Heart
- Epilepsy Research Boosts Case for New Gene Therapy for Dravet Syndrome