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Tag: Gene therapy

World's Most Expensive Gene Therapy Zolgensma Treats Rare Genetic Disease In Infants

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

Doctors Use HIV, Gene Therapy To Cure Bubble Boy Disease

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

Experimental Procedure Could Help Cure Sickle Cell Disease

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

Scientists Having Hard Time Keeping Track Of Patients Who Have Undergone Gene-Editing Treatments In China

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

Promising Gene Therapy Helps Reduce Transfusion In Patients With Blood Disorders

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

Gene Therapy Could Be Key To Survival Of Astronauts In Deep Space Mission To Mars

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

Gene Therapy To Treat Inherent Blindness Will Cost $850,000: Is This The Future Of Medicine?

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

CRISPR And Deafness: Gene-Editing Tool Prevents Hearing Loss In Mice

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

Curing Blindness Through Gene Therapy Might Soon Be Allowed By The FDA

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

Novartis CAR-T Cancer Therapy For Leukemia Gets Landmark Recommendation From FDA Panel

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

How To Beat Cancer? CRISPR Stares Into Its Eyes Then Snips Out The Bad Genes

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

In Depth: How Sickle Cell Anemia Was Cured With Genetic Engineering, According To Doctors

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

Teenager's Sickle Cell Disease Reversed With Help Of Gene Therapy

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

Gene Delivery Therapy Using Supercharged Adeno-Associated Virus Restores Hearing And Balance In Deaf Mice

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

Amgen Researchers Find Gene That May Reduce Heart Disease Risk

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 Debuts With $43.5 Million In Funding

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

Breakthrough Gene Therapy Reverses Vision Loss In Patients With Inherited Blindness

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

EU Regulatory Panel Recommends Approval Of Glaxo's Gene Therapy For Bubble Boy Disease

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

Gene Therapy Shows Potential In Treating Heart Failure

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

Genetic Makeup Of Cancer Tumors May Pave Way For Personalized, More Potent Treatment

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

Suicide' Gene Therapy Triggers Body To Kill Cancer Cells

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

Two Experts Weigh In On 'Suicide Gene Therapy' For Cancer Patients

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

Suicide Gene Therapy And Chemotherapy Combo Kills Prostate Tumor Cells

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

Gene Therapy May Help Restore Immunity In Patients With Rare Immunodeficiency SCID-X1

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

Stem Cell Gene Therapy Shows Promise In Treating Patients With Rare Immunodeficiency Disorder

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

Scientist Call For Moratorium On Human Genome Editing: The Dangers Of Using CRISPR To Create 'Designer Babies'

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

Gene Therapy For Cystic Fibrosis Shows Promise In Mice Study

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

Baby's Leukemia Reversed By New Gene Editing Technique: Therapy May Also Work For Other Diseases

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

Leptin Gene Therapy Could Aid Weight Loss Without Risks Of Bone Loss

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

Spark Therapeutics Gene Therapy Shows Promise In Restoring Vision Of Visually Impaired Patients

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

A Cure For Blindness May Be In Sight Thanks To Vision Cell-Restoring Gene Therapy

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

Gene Therapy To Correct Blindess Can Also Improve Visual Pathways In The Brain

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

Gene Therapy Cures Deaf Mice, Offers Hope Of Treatment For Congenital Deafness In Children

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

Gene Therapy Can Help Treat Cystic Fibrosis Patients

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

Historic Baby In Toronto Born Using IVF Stem Cell Treatment

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

Gene Therapy May Hold Answers For Patients Suffering From Retinal Problems

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

How Blood of Ebola Survivors May Cure Ebola

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

Betting Big on Gene Therapy: Pfizer Partners with Spark Therapeutics for Hemophilia Treatment

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

Will gene therapy be the answer to heart failure? Trial in London starts

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

Gene therapy may lead to biological pacemakers

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

Creating ‘biological pacemakers’ from the heart, a gene therapy breakthrough

"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

Gene therapy used to create biological pacemaker in pigs

A biological pacemaker has been developed in a pig, using gene therapy. Human trials could begin in three years.

Life July 17, 2014

Human gut cells made to produce insulin - hope for type 1 diabetics?

Insulin could be produced by cells found in the human intestine, according to new research from Columbia University.

Life July 2, 2014

Gene therapy may be the wonder 'drug' to cure HIV

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 help cure eye disease choroideremia

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

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