Batten Disease and Gene Therapy

Batten disease occurs when at least one of the thirteen known ceroid lipofuscinosis (CLN) genes is mutated. Gene therapy may offer a one-time treatment to slow or ideally stop the progression of these disorders by delivering a working gene to target the cause of the disease.
The subtitling of these videos was funded in part by a donation from Pfizer, Inc.

[@juliacarroll5040]

Thanks for the video! It makes it easy for those without a medical background to follow. My niece has Batten's ... praying for that gene therapy!! 💜🦋

[@LUIGY-m1b]

How is she doing

[@bigmoneygrip6561]

I hope and pray this works for your niece. I just saw Love, Kennedy and this disease has me in a tizzy. I'm so saddened and truly hope this works. Truly!

[@marisabeltran3084]

😱 omg, it's happening already... congratulation "Jennifer Doudna".

[@bigmoneygrip6561]

Marissa, what's happening already???

[@liamdavis2068]

@@bigmoneygrip6561 What’s happening is it sounds like they’re close to a cure for Barton’s disease.

[@LUIGY-m1b]

Keep up the work on this terrible desisis

[@ollie4999]

My sister has it

[@aaliyahali3314]

@@ollie4999 how is your sister doing she need stem cell and enzyme treatment

[@sorakanzoku1748]

How do I connect with these people we just found out my daughter has a malfunctioning cnl3 receptor and is going to go through this

[@aaliyahali3314]

My son has cln6 and is getting enzyme therapy

[@prabhavikki55]

@@aaliyahali3314Hey. Is he really getting gene therapy? I heard only cln2 has approved gene therapy.

[@RhinoXpress]

This is where AI technology will really excel at treating a disease.

[@adeladel455]

السلام عليكم هل من علاج لهذا المرض

[@noflanofla3789]

السلام عليكم ورحمة الله وبركاته ممكن نتواصل ابنتي مريضة ب cln1

[@adeladel455]

@@noflanofla3789 مرحبا

[@idontknowmuch3441]

No sadly