Comments on: Towards a World without Genetic Diseases

By: My Dream Came True « ScienceRoll

My Dream Came True « ScienceRoll — Wed, 19 Dec 2007 22:47:25 +0000

[...] Health 2.0, Healthcare, Medicine 2.0, Web 2.0, e-Science, science. trackback I’ve already talked about my dream to have a software or service that could help physicians how to find out a diagnosis [...]

By: Genetic Testing: BlogMix and a funny video « ScienceRoll

Genetic Testing: BlogMix and a funny video « ScienceRoll — Thu, 19 Jul 2007 18:25:22 +0000

[...] Towards a World without Genetic Diseases [...]

By: The summary of a long exam period « ScienceRoll

The summary of a long exam period « ScienceRoll — Mon, 09 Jul 2007 18:17:42 +0000

[...] Towards a World without Genetic Diseases [...]

By: Bertalan Meskó

Bertalan Meskó — Mon, 09 Jul 2007 14:50:33 +0000

Dear Gary!

You’ve listed all the usual therapies, but here are some recent improvements on the field. I don’t know how useful these could be:

* Treatment with laxative or NAC reduced mucus accumulation 43% and 50% (http://tinyurl.com/297ccg)

* Emerging drug treatments for cystic fibrosis. (http://tinyurl.com/2z255q)

Unfortunately, we still have to wait for gene therapy: http://tinyurl.com/29lvrx

I hope it helps. It would be my pleasure if you could express your opinion on the following posts:

* http://scienceroll.com/2007/07/01/pseudomonas-infections-and-cystic-fibrosis/

* http://scienceroll.com/2006/11/27/neonatal-screening-in-the-diagnosis-of-cystic-fibrosis/

Thank you in advance!

By: Gary Levin

Gary Levin — Sun, 08 Jul 2007 17:19:41 +0000

Cystic fibrosis, a disease of impaired chloride transport in mucous glands of the pancreas, and lung is a recessive disease with multiple alleles. It is a recessive disease affecting over 20,000 people in the United States. The gene prevalence is 1:2000. Genetic engineering produced Pulmozyme (DNAase) which liquifies the inspissated secretions of the lung, and purified Pancrease (pancreatic enzymes) that improve absorption. There has been tremendous improvement in lifespan, which is shortened by recurring pseudomona infection, severe malabsorption and many other complications.
What is transpring in the area of genetically modifying pseudomonas organisms to be less lethal.
Vectors (adenoviral) carrying the normal gene were ineffective several years ago when inhaled.
Anything else new that you know about?
Thanks (father of son with CF M.D.)

By: iskanbasal

iskanbasal — Sun, 08 Jul 2007 15:53:29 +0000

Thanks for considering my post in your blog, why a genetic defect present at birth….? I also share the hope to be able to treat genetic diseases. It is anyhow a difficult task but as I see there is always a new development and a new achievement. This I think is related to our continuous interest and hope.
iskandar

By: Mendel’s Garden Genetics Blog Carnival #16

Mendel’s Garden Genetics Blog Carnival #16 — Sun, 08 Jul 2007 09:57:37 +0000

[...] Bertalan Mesko at ScienceRoll uses some stunning examples in talking about moving Towards a World without Genetic Diseases. [...]