Dear Dyferliners,

Dysferlinopathies seem to be, at least for instant, a recessive
disorder. Such as the antisense/sense paradigm (suppression of
pathological genes) to treat them would not work. The RNA-mediated
repair of genetic instruction (see the diagram in the Photos section
on the left of this page) seems to be on the contrary (ie at the
manner of Gentamicin principle) particularly well indicated.

Sophareth


--- In dysferlin@y..., "sophareth" <sophareth@y...> wrote:
> Dear members,
>
> Since the dysferlin gene is known, why antisense/sense paradigm has
> not been used more intensively in clinical research? Ce paradigm is
> based on the RNA interference mechanism discovered some 3-4 y ago.
>
> Sophareth Camsonne
>
>
>
>
> --- In dysferlin@y..., Daniel MacArthur <danielm@c...> wrote:
> > Brad:
> > > There are 8 presentations and one discussion workshop on
> dysferlin in
> > > the program for the upcoming Internatioal Congress on
> Neuromuscular
> > > Diseases in July 2002. (one of them is co-authored by group
> member
> > > Daniel MacArthur).
> >
> > Daniel:
> > Thanks for the mention, Brad! While I won't be able to attend the
> > Congress myself, several people from my lab will be paying close
> > attention to the presentations and posters on dysferlinopathies -
> in
> > particular the presenting author of the dysferlin/calpain poster,
> > Harriet Lo, who started her PhD this year investigating the
> diagnosis
> > and pathogenesis of dysferlin- and calpain-related muscular
> > dystrophies.
> >
> > While my research has moved away from dysferlin (I'm now working
on
> > another muscle protein, alpha-actinin-3) I'm still paying close
> > attention to the field of dysferlinopathy - firstly because I
think
> > that understanding dysferlin will greatly illuminate the
processes
> > behind muscular dystrophy in general, and secondly because it's
an
> > extremely interesting and fast-moving field.
> >
> > Brad:
> > > I've listed them on the dysferlin website:
> > >
> > > http://www.dysferlin.org/research.htm#mws2001
> >
> > Daniel:
> > Just a quick question, Brad - I haven't been able to link to your
> > website for several weeks, and I was wondering if you were having
> > any problems with your server, or if it was more likely to be a
web
> > problem at my end?
> >
> > Brad:
> > > Although this research might not have any immediate
implications
> for
> > > patients, the number of research groups giving presentations on
> this
> > > topic is, in my view, a very positive sign.
> >
> > Daniel:
> > I couldn't agree more!
> >
> >
> > Daniel.
> >
> > Daniel MacArthur
> > Institute for Neuromuscular Research
> > Neurogenetics Unit
> > Clinical Sciences
> > The Children's Hospital at Westmead
> > Locked Bag 4001, Westmead
> > Sydney, NSW 2145
> > AUSTRALIA
> > Phone: 61-2-9845-3101
> > Fax: 61-2-9845-3078
> > danielm@c...
> >
> >
> >
>
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