Ride the Storm: Navigating Through Unstable Periods / Katerina Rudko (Belka G...
RM HD SMA220507.pptx
1. The illness
Spino-Muscular
Atrophy (SMA)
A defect in chromosome
5 making *SMN protein
Sadly, after birth the baby
loses more and more muscle
power and eventually
succumbs.
A nerve cell
The science
Click now to see the gene therapy!
No SMN protein made,
nerve and muscle cells
die.
The cell
nucleus with
the DNA
(genetic code)
2. SMN protein is
made and strength
returns!
The illness
Spino-Muscular
Atrophy (SMA)
Sadly, after birth the baby
loses more and more muscle
power and eventually
succumbs.
A nerve cell
The treatment
Click now to see what can happen!
Zolgensma is an
(FDA approved)
gene therapy.
This *AAV9 vector
delivers the
missing gene.