Gene Therapy Explained | Roadmap to a Cure


Decades of research in the gene therapy
field is paying off in 2017. The very first gene therapy products were
approved in the US, with more likely to be approved this year and thousands more
in clinical trials including a trial for Rett syndrome sponsored by AveXis, a
Novartis company, expected to get underway this year so what exactly is
gene therapy it’s the addition of new genes to a person cells to replace
faulty genes in an attempt to prevent improve or cure disease genes need a bit
of help to get into cells one way they get that help is through a virus now
think about what virus is normally do they are really good at getting into our
bodies and making us sick they do that by injecting their genetic material into
ourselves so scientists had the great idea of hijacking a virus to deliver
genes into cells but don’t worry they remove the parts that make us sick the
modified virus is called a vector so how would this work in Rett syndrome well we
know that ret is caused by mutations in a gene called mecp2 which creates a
protein that is very important for brain function
so first billions of copies at the mecp2 gene are manufactured in a lab then the
manufactured genes are packaged inside the vectors in the upcoming clinical
trial a person with Brett will receive a single injection of gene therapy product
called AVXS 201 into their cerebral spinal fluid the vectors will then carry
their precious cargo to the brain and deliver it into the nucleus of the cells
once this is accomplished the vector naturally disintegrates and the new
mecp2 genes go to work making the healthy and vital protein our hope is
that individuals will improve dramatically what symptoms will improve
and by how much and in which individuals these are questions that will remain
unanswered until the clinical trial is undertaken
one thing we do know if improvements do then they won’t happen overnight it will
take time intense therapies and lots of hard work
our SRT started funding gene therapy experiments in 2010 and we’ve spent
almost 7 million dollars to date our first gene therapy consortium launched
in 2014 and generated the data that led to a vexes decision to pursue ret we’re
extremely hopeful but we can’t just sit idle while we wait for trial results
which is why we launched gene therapy consortium 2.0 last year to start
working on a second-generation gene therapy product just in case the goals
of gene therapy 2.0 are to maximize the number of brain cells the vector can
penetrate and also find ways to control the amount of protein the new healthy
genes make the more shots on goal the better which is why we’re also funding a
number of other potentially curative approaches including DNA editing RNA
editing RNA translating and mecp2 reactivation projects just look at what
your donations are making possible together we will leave no stone unturned
on our roadmap to a cure thank you.

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