What We Know About Gene Therapy:
Please click on the links for more information ...
Understanding Gene Replacement Therapy :
Gene therapy is a scientific technique that uses a working gene to treat or prevent diseases.
Gene replacement therapy is a type of gene therapy that uses a “new gene” to replace a faulty or missing gene. This approach attempts to address the root cause of genetic diseases.
Click on the link explore Gene Therapy courtesy of the Columbus Children's Foundation for an explanation of Gene Therapy :
The 2 types of Gene Therapy:
There are 2 types of Gene Therapy currently available for the treatment of AADCd:
USA / Poland : Target Area Substantia Nigra Pars Compacta & Ventral Tegmental
Japan / Taiwan : Target Area Putamen
Both Gene Therapy techniques, delivered using an AAV2-hAADC virus vector to either the Putamen or Substantia Nigra & VTA offer improvements to the severe condition Aromatic Amino Acid Decarboxylase deficiency. The AADC Trust provides support and facts to affected families which are in our possession regarding this invasive but pioneering brain surgery. With this families are able to make the most informed decision as to whether they take advantage of this treatment which is offering a dramatic improvement to severe AADCd symptoms for many.
Target Area Substantia Nigra Pars Compacta & Ventral Tegmental Gene Therapy Statistics:
6 AADCd children have been treated with Gene Therapy (Substantia Nigra Pars Compacta & Ventral Tegmental Area) in the USA.
3 AADCd children have been treated with Gene Therapy (Substantia Nigra Pars Compacta & Ventral Tegmental Area) in Poland.
Considerably more children in Poland are expected to be treated over the next 18 months on a ‘not-for-profit’ basis.
12 more AADCd children will be treated in the USA over the next 18 months, on the ‘Extended Single-Stage Trial’.
This trial is ACTIVE and RECRUITING.
If you are interested in registering for this trial please click on
the link to visit our page "Gene Therapy USA / Poland"....
Please click on the images to discover more about Logan's, Kacper's, Audrey's & Alex's Gene Therapy Journeys:
"It has remarkedly improved his quality of life. He can sleep through the night and actually enjoy life during the day. He is so much happier. He has purposeful movements, which has to be such a neat experience for someone who could never do that. He now has such a better quality of life, which to us was worth the risks of surgery. We would do it all over again if we had to"
Laura (Logan's mum)
"Children have a chance for a new, a better life. I would like for all children with this disease to be able to receive GT treatment and live as close to a normal life as possible, so they are not chained to strollers or beds"
Ela (Kacper's mum)
"It is life changing experience"
"The quality of life improvement for the child is immeasurable and it offers hopes to the child as they continue to improve and develop after the GT"
Ashlee (Alex's mum)
Target Area Putamen Gene Therapy Statistics:
More than 20 AADCd children have been treated with Gene Therapy (Putamen) in Taiwan.
More than 6 AADCd children have been treated with Gene Therapy (Putamen) in Japan.
10 more AADCd children in Taiwan are expected to be treated on a Phase I/II trial during the next 18 months.
This trial is ACTIVE but NOT RECRUITING.
Please click on the images to discover more about Daria's & Satoshi's Gene Therapy Journeys:
"It’s a great happiness not only to continue your children’s lives but to give them a chance to feel, to try and to enjoy a lot more than they can now. I see that GT is a big risk...but a huge piece of happiness you can present to your children is worth it! If you can just do it! Believe in magic!
Viktoriia (Daria's mum)
"I recommend GT. Satoshi can go out longer time on a wheelchair due to the mitigation of the respiratory problems He’s in a good mood and doesn’t cry. He can eat a small amount of food"
Akiko (Satoshi's mum)