Tag: Gene Therapy

Innovative Gene Therapy ‘Reprograms’ Cells to Reverse Neurological Deficiencies

July 14, 2021
Mindzilla

Columbus, OH — A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. The study, led...

New Genetic Copycatchers Detect Efficient and Precise CRISPR Editing in a Living Organism

May 13, 2021
Mindzilla

La Jolla, CA — Researchers at the University of California San Diego have laid the groundwork for a potential new type of gene therapy using novel CRISPR-based techniques. Working in fruit flies...

Gene Therapy Restores Immune Function in Children with Rare Immunodeficiency

May 12, 2021
Mindzilla

Bethesda, MD — An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research...

Gene Therapy Injection in One Eye Surprises Scientists by Improving Vision in Both

December 11, 2020
Mindzilla

Cambridge, UK — Injecting a gene therapy vector into one eye of someone suffering from LHON, the most common cause of mitochondrial blindness, significantly improves vision in both eyes, scientists...

Mimicking the Effect of Exercise with Gene Therapy

December 7, 2020
Mindzilla

Trondheim, Norway — It sounds too good to be true – and it is. But Jose Bianco Moreira and the CERG research group at the Norwegian University of Science and Technology (NTNU) are convinced that...

Scientists Develop New Gene Therapy for Eye Disease

November 27, 2020
Mindzilla

Dublin, Ireland — Scientists from Trinity College Dublin have developed a new gene therapy approach that offers promise for one day treating an eye disease that leads to a progressive loss of vision...

Non-Hereditary Mutation Acts as Natural Gene Therapy in Patient with Rare Disease

November 19, 2020
Mindzilla

São Paulo, Brazil — Researchers affiliated with the Center for Cell-Based Therapy (CTC) in Ribeirão Preto, Brazil, have identified for the first time a non-hereditary mutation in blood cells from a...

Reversal of Glial Scar Tissue Back to Neuronal Tissue Through Neuroregenerative Gene Therapy

November 17, 2020
Mindzilla

Guangdong, China — Brain or spinal cord injury often results in glial scar tissue that is correlated to neural functional loss. Glial scar is a well-known obstacle for neural regeneration due to its...

Editing the Immune Response could Make Gene Therapy More Effective

September 3, 2020
Mindzilla

Pittsburgh, PA — Gene therapy generally relies on viruses, such as adeno-associated virus (AAV), to deliver genes into a cell. In the case of CRISPR-based gene therapies, molecular scissors can then...

Scientists Use Nanoparticle-Delivered Gene Therapy to Inhibit Blinding Eye Disease in Rodents

July 9, 2020
Mindzilla

Bethesda, MD — In experiments in rats and mice, two Johns Hopkins scientists — an engineer and an ophthalmologist — report the successful use of nanoparticles to deliver gene therapy for...

Critical Window for Re-Infection With HIV After Stem Cell Transplantation

May 15, 2020
Mindzilla

Heidelberg, Germany — Some people with HIV need to undergo an allogeneic stem cell transplantation in order to treat different types of blood cancer. Most of the patient’s immune cells are eliminated...

Study Shows Glaucoma Could Be Successfully Treated With Gene Therapy

April 22, 2020
Mindzilla

Bristol, UK — A new study led by the University of Bristol has shown a common eye condition, glaucoma, could be successfully treated with a single injection using gene therapy, which would improve...

Simulations Show How to Make Gene Therapy more Effective

April 20, 2020
Mindzilla

Groningen, Netherlands — Diseases with a genetic cause could, in theory, be treated by supplying a correct version of the faulty gene. However, in practice, delivering new genetic material to human...

Gene Therapy Generates New Neurons to Treat Huntington’s Disease

March 3, 2020
Mindzilla

Guangdong Province, China — Huntington’s disease (HD) is a rare disease characterized by abnormal chorea movement and caused by Huntingtin (Htt) gene mutation and neurodegeneration in a brain...

Gene Therapy/Gene Editing Combo could Offer Hope for some Genetic Disorders

February 13, 2020
Mindzilla

Washington, DC — A hybrid approach that combines elements of gene therapy with gene editing converted an experimental model of a rare genetic disease into a milder form, significantly enhancing survival,...

Novel Techniques for Mining Patented Gene Therapies Offer Promising Treatment Options

February 10, 2020
Mindzilla

West Lafayette, IN — The global gene therapy market is expected to reach $13 billion by 2024 as new treatment options target cancers and other diseases. Now, a team of scientists from Purdue University...

Six Patients With Rare Blood Disease Are Doing Well After Gene Therapy Clinical Trial

January 29, 2020
Mindzilla

Los Angeles, CA — UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked...

Nanoparticles Deliver ‘Suicide Gene’ Therapy to Paediatric Brain Tumours Growing in Mice

January 9, 2020
Mindzilla

Baltimore, MD — Johns Hopkins researchers report that a type of biodegradable, lab-engineered nanoparticle they fashioned can successfully deliver a “suicide gene” to paediatric brain tumour...

Researchers Solve Protein Structure Associated with Inherited Retinal Diseases

January 9, 2020
Mindzilla

Durham, NH — Researchers at the University of New Hampshire have reported the first structural model for a key enzyme, and its activating protein, that can play a role in some genetically inherited...

Gene Therapy Shown to Offer Long-Term Benefits for People with Haemophilia A

January 7, 2020
Mindzilla

London, UK — A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in the UK. Clinical researchers...

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