The Food and Drug Administration has approved the use of Zolgensma in treating a rare genetic disease in infants. At a one-time treatment, Zolgensma costs at least $2.1 million.
Medicine May 25, 2019
Researchers developed a new way to treat SCID and doctors call this a cure. Children who received the new therapy are now producing the type of cells needed to fight infection.
Public Health April 17, 2019
Researchers reported that a patient who has gone through gene therapy to cure sickle cell disease no longer experiences symptoms and has an increased level of fetal hemoglobin. The researchers will continue clinical trials before offering it to the public.
Medicine January 28, 2019
While gene therapy is yet in its early stages, scientists need to keep tabs of patients who have received gene-editing treatments. However, scientists in China were not keeping track of patients who had the experimental gene therapy that may trigger health problems later on in life.
Biotech December 30, 2018
People with beta-thalassemia, a genetic blood disorder, could benefit from a promising gene therapy. The therapy, LentiGlobin, is reported to reduce or eliminate the need for blood transfusions in patients enrolled in Phase 1 and 2 trials.
Public Health April 19, 2018
High-dose radiation in space could be deadly for astronauts on a mission to planet Mars. What are ways through which gene therapy can increase their odds for survival?
Space April 8, 2018
Luxturna, a drug for gene therapy to cure inherent blindness that was recently approved by the FDA, will cost $850,000. Does the prohibitive cost outweigh the advantages of a one-time treatment to cure a hereditary disease?
Medicine January 4, 2018
Scientists recently cured mice with progressive hearing loss using a new genetic engineering technique. What is the CRISPR-Cas9 and how could it cure deafness?
Public Health December 30, 2017
Patients whose blindness is a result of an RPE65 gene might soon consider Luxturna, a form of gene therapy that improves vision. Its approval will soon be deliberated by the U.S. Food and Drug Administration.
Biotech October 10, 2017
A new gene therapy developed by Novartis has received crucial recommendation from an FDA advisory panel on July 12. Experts believe the treatment’s approval will pave the way for new gene-based medicines.
Medicine July 13, 2017
Scientists used the CRISPR tool to replace cancer-causing fusion genes with cancer-fighting genes in mice. This experiment could pave the way for the gene-editing tool to be used against cancer in the future.
Biotech May 5, 2017
For the first time, doctors have claimed that sickle cell anemia is curable with gene therapy. Doctors at the Necker Children's Hospital in Paris, who treated a teenager with the blood disorder, claimed complete cure after gene therapy.
Public Health March 5, 2017
A teenage boy's sickle cell disease was reversed after gene altering therapy. While still not considered a cure, scientists are optimistic about the new therapy's merits in the search for a cure.
Medicine March 2, 2017
A new gene delivery therapy using supercharged adeno-associated virus restored hearing and balance in deaf mice, offering hope to about 30 million people in the United States suffering from hearing loss. How does the treatment work?
Biotech January 30, 2017
DeCODE genetics, an Amgen-owned company, claimed it has identified a new genetic variant involving the ASGR1 gene, a finding that can reduce heart disease risk by 35 percent.
Medicine May 19, 2016
Gene editing startup Homology Medicine raises $43.5 million in its Series A funding. The company hopes to use naturally occurring genetic mechanisms to cure rare diseases.
Biotech May 4, 2016
Gene therapy fuels hope for slowing or reversing loss of vision in patients with choroideremia, a rare form of inherited blindness. The trial, a first of its kind in the world, saw sustained benefits in its patients.
Healthy Living/Wellness April 30, 2016
An EU panel has recommended the approval of a Glaxo gene therapy for Bubble Boy Disease. The treatment gives hope to patients with the rare disorder that currently does not have a medicinal treatment.
Life April 2, 2016
A new gene therapy that could potentially improve a heart failure patient's cardiac function has yielded promising results. Experts believe the treatment is the 'ultimate method' to reconstruct damaged heart tissue.
Life April 1, 2016
The genetic makeup of tumor may hold the key to personalized and more potent cancer treatment. A new research found the so-called 'Achilles heel' of cancer cells, which future treatments can target.
Life March 4, 2016
Scientists were able to successfully kill prostate cancer cells using suicide gene therapy. The implication of the new discovery may also provide promise to the way all cancers are treated.
Life December 15, 2015
A new gene therapy has been developed in which a virus is injected into a cancer cell. The technique causes the tumor cell to self-destruct, alerting the immune system to attack other cancer cells.
Life December 14, 2015
Survival rates of prostate cancer patients treated with suicide gene therapy were found to be five to 20 percent better. The therapy involves modifying the prostate cancer cells to trigger the body's immune system to attack them.
Life December 13, 2015
Researchers make a breakthrough in treating X-linked severe combined immunodeficiency. Older kids and younger adults could have their immune systems built up with gene therapy.
Life December 8, 2015
Gene therapy appeared beneficial in X-linked severe combined immunodeficiency (SCID-X1). Typically affecting men, Bubble Boy disease is a rare genetic disorder caused by IL2RG gene mutations.
Life December 7, 2015
Gene editing biotechnology known as CRISPR-Cas9 lets scientists alter genes in animals and plants rapidly and inexpensively. Experts expressed worry about the risks and potential abuse for this human gene editing tool for creating "designer babies."
Life December 6, 2015
Previous attempts to use gene therapy in treating cystic fibrosis were largely uneffective. But after seeing gene therapy used to successfully treat genetic disorders like hemophilia, scientists reattempted and succeeded in curing the disease in mice and human cell cultures using the approach.
Life November 17, 2015
A previously untested gene editing technique was able to save the life of 1-year-old Layla after she was diagnosed with leukemia. Researchers are continuously researching to refine the technique so that it can be used to treat other gene-related disorders.
Life November 9, 2015
Weight loss treatments almost always entail bone loss. Now, researchers may have finally found a weight-cutting regimen that does not risk bone health: leptin gene therapy.
Life October 24, 2015
Gene therapy that aims to restore vision in partially blind people exhibited positive results in its phase 3 clinical trial. Experts said that the results were promising and that patients were able to achieve the maximum benefits set for the drug.
Life October 7, 2015
By inserting a human gene for a light-sensing pigment, researchers were able to restore some vision in mice whose vision cells were destroyed. The work suggests that a similar strategy might succeed in humans.
August 18, 2015
Researchers showed that not only is gene therapy capable of restoring sight but it can also spur brain connections back into action despite being inactive for a while.
Life July 22, 2015
Mice that had defective genes replaces with working copies showed evidence of a restored ability to sense sound, researchers say. The finding could help with human congenital deafness, they suggest.
Life July 8, 2015
Cystic fibrosis may, one day, be treated by replacing faulty genes responsible for the disorder. Does this mean a treatment for this deadly condition is around the corner?
Life July 5, 2015
A revolutionary IVF treatment known as Augment has assisted a couple in Toronto in having their first baby. Eight more women are expected to deliver Augment newborn babies.
Life May 8, 2015
Retinal problems have always led to vision loss, but researchers are now saying gene therapy can prevent such, used as a means of treating Leber congenital amaurosis.
Life May 4, 2015
Rick Sacra survived Ebola, now his blood may provide the raw material to develop a new generation of drugs to battle the disease.
Life December 22, 2014
Gene therapy has seen disappointments and safety concerns in the past but Pfizer is establishing a gene therapy platform and is working with biotech company Spark Therapeutics to develop a treatment for Hemophilia B. Here's why.
Life December 10, 2014
Scientists from Britain will conduct a clinical trial to assess a gene therapy that could potentially replace the need for heart transplant in patients with advanced heart failure.
Life August 12, 2014
Researchers developed a method that could transform ordinary heart muscle cells into biological pacemaker, a development that could lead to the replacement of electronic pacemakers in the future.
Life July 20, 2014
"Biological" pacemaker could replace electronic version in humans, researchers say. Gene therapy can create alternate "timekeeper" for the heart, they report.
Life July 17, 2014
A biological pacemaker has been developed in a pig, using gene therapy. Human trials could begin in three years.
Life July 17, 2014
Insulin could be produced by cells found in the human intestine, according to new research from Columbia University.
Life July 2, 2014
Doctors are interested in gene therapy for treating HIV as there are some people who have natural immunity to the virus. A study conducted by a team of researchers shows that genome editing can be a safe treatment option for HIV.
Life March 8, 2014
Gene therapy may hold the solution for patients suffering from choroideremia, a rare form of progressive blindness, as researchers find that it's possible to improve vision after injecting the eye with the missing or defective gene.
Life January 16, 2014