TERAPIA GENETICA
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
La terapia genica è un approccio rivoluzionario al trattamento delle malattie genetiche, in cui un trattamento una tantum può fornire benefici per tutta la vita. Invece di trattare solo i sintomi, la terapia genica risolve la malattia alla fonte sostituendo il gene difettoso con una copia sana.
Una copia sana del gene difettoso del paziente viene caricata in un virus che è stato privato del proprio DNA.
Trilioni di virus, ciascuno contenente una copia sana del gene, lo sono iniettato nel liquido spinale del paziente.
I virus si legano alle cellule del midollo spinale e del cervello del paziente e forniscono geni sani al nucleo della cellula.
come funziona
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
come funziona
i nostri partner
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!