Gene Therapy - 6 stories for patients with genetic disease

6 stories about gene therapy - at the heart of each story lies a technology or a regulatory step that affects many genetic disorders.

We cover in this video medications that we tailored specifically for nonsense mutation, missense mutations, splice site mutations, and a gain of function mutations, as well as technologies that are not mutation specific and can affect the entire gene.

If your mutation type is nonsense, we invite you to join us at the nonsense mutation foundation. By grouping up, we can broaden the usage of nonsense mutation medications to treat patients across many genes. Visit us at:

We wish you good health, no matter what is your disorder. 😘
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Special Thanks:
We would like to thank to the Nisnkorn Family for sharing Hodaya's story.
We would like to thank to Prof. Hagit Baris Feldman and Lynne Maquat for assisting with the text behind this video, and escorting us in the creation process.

The video was narrated by Alden Schoeneberg,

Sound and video license:
The music in this video came from a range of sources:
- YouTube sound library

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▶️ Other video:
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#GeneTherapy #NonsenseMutationFoundation #GeneticDiseases #nonsensemutation #rarediseases #geneticmutation #geneticdisorders

[sauce]

I have twin babies.. identical.. both with PRRT2 deletion. Does this help? ASO?

[mrjuttcars]

Sir when will Becker muscular dystrophy be tretead with this tharapy please

[coolsrilwan]

Is any gene therapy for GRIN2B DISORDER??

[SnowWhite-xlnr]

My son has Fragile x.. Can gene therapy help him?

[samyn]

Is there any treatment for depression?

[laxmiadisachin]

My son suffering from dmd exon 48to exon 52 gene is skipping plz any cure treatment is plz help me

[rizwanrehman]

Is the gene therapy is better for prepionic acidemia. Plz rep must.my baby is suffering from this rare disease

[ravikishorevemuri]

Neurogenic bladder & bowel due to spina bifida
will gene therapy works ??
please let me know my two years daughter is suffering

[fatimanadaf]

Is it possible for Autosomal Recessive gene.. Disease is diagnosed as " Salt and pepper developmental regression syndrome"

[ankitakothari]

My cousin brother is diagnosed with DMD muscular dystrophy having 53 rd gene skipping. With the help of gene therapy is there any treatment of this disease.. plz reply

[ajaymeena]

Is gene therepy possible to cure polyctic kidney disease 🤔

[JeffreyJorge-pp]

I'm schizophrenic I was wondering if scientist could answer this question for me why can't I work and being they are working on enhancing medication will they ever medication to address that fact ....I wonder would they make a pill where a schizophrenic person can become fully functional