"We focused on the eye because we thought reversing blindness would be pretty cool and could be a drug, a very quick drug to develop relative to the whole body. And it worked."
"The future of gene mutation is here. Gene therapy, which is the thing that's going to bust open longevity, is here."
"Over time, it’s possible that gene therapy could be as common as chemo and radiotherapy in treating genetic cancers."
"We can put backup copies of that mitochondrial DNA into the nucleus, into the nuclear DNA with regular gene therapy."
"His body still has to catch up and every day he's working on signaling that gene."
"The very first gene therapy that was actually approved to go in and modify a living person's genes to cure a genetic disease."
"We could give you gene therapy right now and change your fiber type composition almost surely."
"Crisper and Gene therapies are minimizing severe diseases."
"Gene therapy marks a revolutionary approach to addressing diseases by targeting their underlying genetic causes."
"Gene therapy... could rewrite those wrong instructions... like catching a design flaw early before countless defective products get manufactured... introduces functional genes into a patient's cells to compensate for the flawed disease-causing versions."
"My project is really to develop a new treatment for dopamine transporter deficiency and using gene therapy to deliver a normal copy of the gene as a new treatment."
"A one-time gene therapy could theoretically lead to lifelong production of medication within the eye and basically free patients from that treatment burden of monthly injections."
"Treatment, a gene therapy for LCA... is the first FDA approved treatment, gene therapy treatment in the eye and not just in the eye, in children."
"That's the beauty of gene and cell therapy."
"Gene therapy is ethical because it can be supported by the fundamental moral principle of beneficence. It would relieve human suffering."
"Patisserin, a TTR Gene silencer, can address the high unmet need for patients with ATTR cardiomyopathy."
"We are just on the cusp of hundreds of companies working on CRISPR and gene therapy."
"Gene therapy, treatment of human disease by transferring genetic material into specific cells of interest."
"We can use the gene therapy technologies for vaccine purposes; we can turn the whole thing on its head."
"Gene editing works not only in rare diseases but in more complex diseases like diabetes and even cardiovascular disease."
"Gene therapy represents a paradigm shift in disease treatment, offering a novel approach that targets the fundamental genetic causes of ailments."
"If we understood how the genes in a larval honeybee are modified in different directions to produce a queen or a worker, we would be much closer to using gene therapy to cure human diseases."
"Oxygen under pressure is a drug, and it's a drug that turns on off up to 6,000 genes in your body."
"Stem cell therapy and gene therapy trials are going on."
"This will be among the first viral gene therapies that's going to be licensed in the next five years or so."
"Viral gene therapy uses the vectors, the genomes of viruses we call them viral vectors, in a number of different ways."
"Using viral vectors has many, many benefits."
"Gene therapy is a process where they modify your cells, the DNA in your cells, to correct genetic defects."
"We can also reverse it and get the cells to wake up and remember how to read the youthful pattern of genes that they did 50, 60 years ago."
"You can change the way that gene is going to express itself using gene editing and cell therapy."
"Germ line gene therapy is when you alter the DNA in a gamete so that the offspring would not inherit the faulty allele."
"Gene therapy has given me the ability to take full control of my life."
"Gene therapy is an additional option that can cure SCD and is more accessible."
"It's very important that we support gene therapy because it's a lot of people like me who want to be relieved of this pain."
"You're using gene editing and cell therapy combined and you're actually curing or reversing disease."
"We're going to be curing cancer, we're going to be curing genetic diseases."
"This is actually the first very successful use of gene therapy."
"Born completely deaf, a young girl can hear now after getting groundbreaking gene therapy."
"It's a game changer. We can start to use gene therapy in young children to restore hearing."
"The virus has the interesting property of being able to be retrogradely transported back to the cell body, find the TH gene, remove the Neo, and turn TH back on."
"In your lifetime, you are going to receive a viral gene therapy as medicine."
"The vision back in 2008 was, could we reinvent the gene vaccine and develop a completely synthetic vaccine approach that didn't have the complications of cell culture?"
"Gene therapy is a mechanism by which genetic disorders can be cured or treated by masking the effect of the faulty allele."
"The drug's an example of what's called gene silencing."
"One of the major benefits of these gene therapy platforms is the advantages in discovery and development over traditional means of developing drugs."
"The effectiveness of this treatment highlighted the significant impact that a gene therapy can have for patients where no other treatment option is available."
"Our website currently hosts tons of reliable resources that educate on gene and cell therapy and are free to share."
"Gene therapy is simply put, an introduction, removal, or change of genetic material within the patient's cells."
"Gene therapy is the future of medicines."
"At CCF, we focus on the acceleration of the most effective gene therapy treatments for patients with ultra-rare diseases."
"Our objectives are to share about AAV, why we're excited about it, dive a little deeper into the challenges that you hear about."
"In researching and understanding AAV, we've shown through many different animal studies and in clinical studies in humans that there is a strong safety profile for AAV."
"We're excited about what AAV can do, what it has been able to do."
"These guys have literally a 95% market share in making proteins that are used to make vaccines and are used in gene therapy."
"We're continuing to further improve on the system, make it more specific, more efficient, develop delivery methodologies so that we can use it to edit cells in a very profound and efficient way in the body."
"Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited."