Personalized care:
Every gene therapy plan is as individual as you are. Our experts tailor treatment to your diagnosis, genetic makeup and needs, providing care that’s precise and focused on you.
Did you know your genes can hold the key to life-changing treatments?
Understanding how genes work is unlocking new possibilities for treating serious inherited conditions. Gene therapy can target the root causes of certain genetic blood disorders, as well as neurological and eye conditions. And it offers new treatment opportunities for previously incurable diseases. Gene therapy (or interventional genetics) is an emerging field, and one that is seeing a great increase in federally approved treatments and research.
Our gene therapy program provides specialized care for children with genetic conditions and adults with certain cancers and blood disorders. You’ll have expert guidance and support as you explore this promising path — one that uses the power of your own genetic code.
Every gene therapy plan is as individual as you are. Our experts tailor treatment to your diagnosis, genetic makeup and needs, providing care that’s precise and focused on you.
Our experts use approved gene therapies and clinical trials to bring you the latest treatments. As part of the important work happening in biologic medicine, we continue to be at the leading edge of available therapies.
Our experienced pediatric team supports children and their families throughout their gene therapy journey, while adults receive care tailored to their unique needs.
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Some appointments still need to be in person but virtual visits let you check in from home for quick follow-ups or when travel isn’t easy.
When you or your child has a genetic condition, your genes change and stop making the proteins your body needs — or they make the wrong ones. A faulty gene is often the underlying cause of inherited health problems.
Gene therapy can help by replacing, repairing or turning off the gene that’s causing the issue. This change can safely address the root cause of disease, but it won’t affect the inheritable part of your DNA. While not all conditions respond to interventional genetics, it works best for those caused by changes to a single gene.
We currently offer gene therapy for these conditions:
We use gene therapies (including Casgevy™, Lyfgenia™ and Zynteglo™) and cell-based therapies to treat several inherited and acquired blood disorders, including:
Our team uses the first U.S. Food and Drug Administration (FDA)-approved gene therapy (Otarmeni™) designed to treat genetic hearing loss. The therapy is approved for both children and adults who have severe-to-profound deafness. Variants in a certain gene (the OTOF gene) that controls auditory neuropathy cause this. The therapy delivers a functional copy of the OTOF gene directly into your inner ear.
You may be able to have gene therapy if you have an inherited retinal disease (retinitis pigmentosa). The FDA has approved one gene therapy for this eye disease for people with mutations in both copies of a certain gene (the RP65 gene). Our researchers are also studying other therapy alternatives in ongoing clinical trials.
Gene therapy also offers new hope by addressing underlying causes of certain genetic conditions affecting muscles and movement, like:
Gene therapy (Skysona™) may also help conditions like adrenoleukodystrophy (ALD), a genetic disorder that affects your nervous system and adrenal glands.
We build your care team based on your inherited condition, bringing together providers experienced in genetics, diagnosis, treatment and long-term support. Because gene therapy can involve many areas of medicine, your team is tailored to your unique needs. Your providers work together across specialties to make sure you receive comprehensive, seamless care.
We offer gene therapy at the following location in Ohio.
No two gene therapy plans are the same because everyone’s genetic makeup is different. Some therapies use modified stem cells from your blood. Others use lab-made genes. Once introduced to your cells, these modified genes then grow and replace damaged or missing genes. You may have several treatments. And some may require a hospital stay, while others may not.
Before treatment, you may need genetic testing and other blood tests. We may also recommend chemotherapy. If your treatment involves stem cells, you’ll need a procedure called bone marrow aspiration, where we collect bone marrow (and the stem cells inside it) from your hip bone.
Our team personalizes care to your needs and explains what to expect before treatment begins.
Currently, we use CAR T-cell therapy to treat blood cancers, like lymphoma and multiple myeloma. And we use gene-editing tools to better treat inherited blood conditions, like sickle cell disease and beta thalassemia. Our neurology, otolaryngology (ENT) and ophthalmology teams also use gene-based treatments to improve care.
Not every treatment is right for everyone, but gene therapy may be an option worth considering. As the field of interventional genetics advances, our experts are helping patients navigate today’s approved therapies while looking ahead to emerging innovations. We’re here to provide guidance, answer your questions and help you understand the opportunities that may be available.
Getting an appointment with one of our gene therapy experts is easy. We’re here to help you get the care you need.
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