TERAPIA DE GENES
HELP US REACH OUR CURRENT $1.5M GOAL!
Your support is crucial in raising awareness and funds for gene therapy pre-clinical research, preparation for a Toxicology Study, or whatever exciting initiatives may be next.
The sooner we reach our goals, the faster we get to a potential Clinical Trial.
See our Fundraising Progress below.
Thank you for your SUPPORT
A terapia genética é uma abordagem revolucionária para o tratamento de doenças genéticas, onde um tratamento único pode proporcionar benefícios para toda a vida. Em vez de tratar apenas os sintomas, a terapia genética corrige a doença na origem, substituindo o gene defeituoso por uma cópia saudável.
Uma cópia saudável do gene defeituoso do paciente é carregada em um vírus que foi despojado de seu próprio DNA.
Trilhões de vírus, cada um contendo uma cópia saudável do gene, são injetado no fluido espinhal do paciente.
Os vírus se ligam às células da medula espinhal e do cérebro do paciente e entregam genes saudáveis ao núcleo da célula.
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the gene therapy project
Several years ago, a group of ECHS1 families forged a partnership with researchers in the Bailey Lab at UT Southwestern Medical Ceneter, located in Dallas, TX, USA.
The Bailey Lab is working on the use of an adeno-associated viral (AAV) vector for therapeutic gene delivery.
the bailey lab team
Dr. Rachel Bailey is an Assistant Professor in Pediatric Neurology with a secondary appointment in the Center for Alzheimer's and Neurodegenerative Diseases.
Meghan Eller is a Neuroscience Graduate Student and
Madison Bunce is a Green Fellow.
the teaM AT WORK
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nossos parceiros
It truly takes a village.
Families from around the world are partnering with Cure Mito Foundation to raise funds that get us closer to a treatment or cure for our children.
If you are interested in supporting a fund which honors a child or family member affected by ECHS1 Deficiency, please click the "DONATE" button associated with one of the campaigns below.
Please email info@curemito.org if you would like information about starting a fund in honor or memory of your loved one.
Thank you for supporting our children!
