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#6345 - 11/23/06 02:48 PM Ichthyosis advances
threerxli Offline
Member

Registered: 01/14/03
Posts: 515
Loc: virginia
Hello everybody.

The last issue of "Nature Medicine" published a report on the first
successful cure of a rare genetic skin disease case.

The patient was affected by Epidermolisis Bullosa (EB) and part of his
skin has been (apparently permenently) repaired with the correction of
the genetic mutation causing the pathology.

Stem cells have been used, with a modified virus as carrier. The
experiment was carried out in Modena, Italy.

More info as soon as I can. The methodology can apparently be used to
cure also other kinds of genodermatoses as Lamellar Ichthiosis. It will
take time, but it seems to be the first real success!

Ciao, Flavio

I guess that I forgot to add that this was emailed to me...

Darcie


[This message has been edited by threerxli (edited November 27, 2006).]

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#6346 - 11/23/06 10:08 PM Re: Ichthyosis advances
Kotick Offline
Member

Registered: 01/29/06
Posts: 205
Loc: Brisbane, Australia
WOW! Sounds interesting...

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#6347 - 11/23/06 11:30 PM Re: Ichthyosis advances
Nana-1 Offline
Member

Registered: 07/10/06
Posts: 66
Loc: Cleveland, Ohio, USA
WOW - WOW - PLEASE KEEP US POSTED. WONDERFUL NEWS.

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#6348 - 11/24/06 12:18 AM Re: Ichthyosis advances
CShell Offline
Member

Registered: 11/10/05
Posts: 1193
Loc: Fort Meade, MD
WOW that's fantastic!!! I want to hear more!!!
_________________________
Courtney
Mom to Julia

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#6349 - 11/25/06 01:47 AM Re: Ichthyosis advances
gryphon Offline
Member

Registered: 04/12/06
Posts: 298
Loc: Alberta, Canada
That's interesting. Thanks for the info, Flavio. I remember research as far back as the mid 90's re: gene therapy and LI. So a breakthrough for LI may be not too far off.

What I also find interesting is how much of the recent advancements stem-cell research is taking place in Europe and Asia. Just this week, the national media was lauding the use of stem cells to prevent, and even reverse, muscle wasting in the Dechenne (sp?) type of Muscular Dystrophy. There too it was an Italian institute that undertook the research. The research was published in the regular journal Nature this month.

I do wonder though to what extent both the increasing restrictions placed on stem cell research by lawmakers in the US, and the special-interest pressure to handcuff researchers are delaying the advancement of gene therapy for ichthyosis in the US. Europe now even seems to be where many US researchers working.

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#6350 - 11/26/06 04:07 AM Re: Ichthyosis advances
pauline5 Offline
Member

Registered: 01/06/02
Posts: 913
Loc: Melbourne, Australia
This is amazing news for EB sufferers, i can't think of a more deserving group of people to get this potential relief of pain...such a terrible condition to have to endure on a daily basis...

I am sure that KatieCakes will be deliriously happy about this, as she has made many friends with EB over the years...

Well done medicos...

Pauline

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#6351 - 11/26/06 04:31 PM Re: Ichthyosis advances
gryphon Offline
Member

Registered: 04/12/06
Posts: 298
Loc: Alberta, Canada
When I first read this thread, I thought of Jonny Kennedy and all his efforts to raise funds and awareness for EB research. As Pauline wrote, it will be a great achievement if it helps to reduce the pain and difficulties those with EB have to endure.

Having IV, I don't use retinoids and so I can't comment on its history in the US as Tatiana did. But I spend quite a bit of time in the US and can say that even with lotions in general, there is much less selection OTC as compared to what can be found in the Canada. Whether it is lotions with Urea, Na-PCA or hyaluronic acid, it is difficult to find the same selection on store shelves. Can't say why that is. In the 80's I switched from lactic acid to urea in my primary lotions. Most of my extended family are Americans and whenever I spent time back then in the US, I had to travel with the lotions as I could never find urea lotions OTC in US stores whereas there were several lotions in Canada that I could buy at any drugstore. European cream availability in the US is similar. European lotions are often of exceptional quality although very expensive. As examples, Dexeryl, Lipikar and Exomega can be found in Canadian stores but the same can't always be said in the US.

Now a little bit of soapbox time for myself. On the topic of gene therapy, I'm troubled by how stem cell research is tied to the abortion issue in the US. As I understand it, embryonic stem cell research can be undertaken even w/o the "harvesting" of stem cells from fetal abortions. There is sufficient supply just from the abandoned zygotes at fertility clinics and miscarriages at hospitals. Those sources of stem cells are currently destroyed with no religious thought or ceremony. So I wonder why the embryonic stem cell research in the US can't be unfettered and kept apart from the whole debate on abortion. I sense that the full potential of ichthyosis gene therapy efforts in the US is restricted as a result. As I see it, there is tremendous capacity for achievement in the US but so much financial, institutional and human capital is being held back in this field when compared to Europe and Asia.

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#6352 - 11/26/06 05:21 PM Re: Ichthyosis advances
aylasmom Offline
Member

Registered: 05/29/06
Posts: 215
Loc: Waterford ,MI
We banked Ayla's cord blood (stem cells) because I had Hodgkins lymphoma when I was younger and we figured better safe than sorry...

We were told that from the small sample they can generate as much a necessary if we ever needed it-

I was also told that people can donate cord blood to research purposes after they deliver their baby.

We didn't donate ours, because we wanted it for our own safety net. Although I would be happy to donate a portion to Ich research if it was needed-

please feel free to correct me if I am wrong on the following, I am still learning...

I recently did a project for my microbiology class in which I researched Adenovirus (This is one of the viruses they often use to deliver the corrected gene) and gene therapy. I think its important to mention that although inactivated, viruses can still mutate to regain pathogenicity. This is one of the reasons that gene therapy is not more widely used. In some test cases (not Ich related), although symptoms of the original condition were alleviated, people developed other illnesses as a result of the gene therapy and in some cases even died! Sorry to be a downer...I just think its important to note.

I think the advancement mentioned in the first post is great news!!! Are there websites that you can translate to English on? I would like to read CuteLawChicks post, but don't know how- Thanks for sharing...

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#6353 - 11/26/06 06:21 PM Re: Ichthyosis advances
Jr88 Offline
Member

Registered: 03/13/04
Posts: 36
Loc: Vancouver, BC, Canada
I decided to skip the process of explaining how to translate the text above, and do it for the board.

According to Babelfish translation (http://babelfish.altavista.digital.com/babelfish/tr) ...( rough translation, I might add)

Genic therapy cures a serious genitica disease of the succeeded Important skin the first clinical study on a sick patient of a shape of bollosa epidermolisi * (EB), devastating genetic disease of the skin that provokes the separation of the superficial layer, the epidermide, from that deepest one, the derma. After a year from the treatment, the appraisal of the result in the zones transplanted has confirmed complete regeneration and maintenance of the "new" epidermide, without development of bollose lesions. The job, published on authoritative the Natures Medicines *, is work of a group of investigators coordinates to you from Michele De Luca, ordinary university professor of biochemistry near the Department of Biomedical Sciences of the University of the Studies of Modena and Reggio Emilia, Scientific Director of Foundation Bank of the Eyes of the Veneto, and from Fulvio Mavilio, ordinary university professor of Molecular Biology, Faculty of Bioscienze and Biotecnologie, University of the Studies of Modena and Reggio Emilia. Both have received numerous Telethon financings to leave from 1993. An adult of 36 years, hit from the not lethal shape of bollosa epidermolisi, giunzionale call (JEB), is enlisted for a said clinical experimentation of phase I/II: a study pilot in which for before it turns a new treatment comes somministrato to a patient in order to try to cure one disease. The result achieved in this phase concurs to draw indications approximately the effectiveness of the treatment and, in positive case, it supplies elements for the just doses and the drawing up of the relati to you protocols of somministrazione. The disease depends on a defect in the gene for the chain beta 3 of laminina 5, a protein that allows that epidermide the attacks to derma and without which the skin detaches with consequent inflammation and infection. For before the time, coming from staminali cells from the epidermide of the patient, captured with a cutaneous biopsy, have been genetically corrected by means of the insertion of a retrovirale carrier of the healthy version of "the sick" gene (genic therapy), and made to grow in laboratory. The increase has been continuation until obtaining nine borders of epidermide, sufficiently large from being able to be transplants to you on two zones of the legs of the sick one. "the priority comes more given to those zones to risk or more susceptible than infections, but the possibility exists to replace l?epidermide nearly all the parts of the body" - Michele De Luca explains - "the taking root has been completed to eight days from the transplant and, after a year, epidermide of both the legs dealt have maintained to a normal aspect and normal property of adhesion". The maintenance of the new epidermide depends on the presence of staminali cells genetically corrected that they guarantee I renew of the woven one. "It is before the time - De Luca continues - than a genetic disease of the skin comes cured thanks to an participation that joins the genic therapy to the cellular therapy. After to have concluded this clinical study, the next step will be to extend this treatment to other genetic diseases of the skin, like the various shapes of EB or the ichthyosis to lamellare, and to diseases of other epithelia, like as an example the corneali dystrophies, invalidating diseases that they carry to blindness ". "We are of forehead to the first one succeeding of a protocol of genic therapy to outside of the ematopoietico system, that is of the cells of the blood, and that it uses epiteliali staminali cells" comments Fulvio Mavilio, molecular and expert biologist of retrovirali carriers that just this year have obtained a Telethon financing for two years on the interactions between retrovirali carriers and human genome. The sophisticated technique to the base of the genic therapy has been put to point from Graziella Pilgrims, associate university professor of Cellular Biology near the University of Modena and director of the Laboratory of the Center Searches on the Cells Staminali Epiteliali di Fondazione Bank of the Eyes of the Veneto. A contribution to the search comes also from Clear Bonini, ricercatrice of the Saint Raffaele of Milan that has studied the immunological aspects of the treatment. The clinical part of the therapy has been continuation from Alberto Giannetti, Clinical director of Dermatological of the University of Modena and the Integrated Medicine Department and the Medical Specialty dell?Azienda Ospedaliero-Universitaria of Modena. The study, left many years ago, has been mainly financed from Telethon. Other financings are joints from the AFM (Association Franšaise contre les Myopathies), from the European Community "Plan Skin Therapy" YOU Program Picture, from the University of Modena and the Foundation Bank of the Eyes.

Kind Regards
Jason

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#6354 - 11/26/06 06:32 PM Re: Ichthyosis advances
aylasmom Offline
Member

Registered: 05/29/06
Posts: 215
Loc: Waterford ,MI
Thanks Jason!!! I'm not very internet savvy... I really appreciate the help!

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