Asset Details
Do you wish to reserve the book?
Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD
Hey, we have placed the reservation for you!
By the way, why not check out events that you can attend while you pick your title.
Oops! Something went wrong.
Looks like we were not able to place the reservation. Kindly try again later.
Are you sure you want to remove the book from the shelf?
Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD
Do you wish to request the book?
Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD
Please be aware that the book you have requested cannot be checked out. If you would like to checkout this book, you can reserve another copy
We have requested the book for you!
Your request is successful and it will be processed during the Library working hours. Please check the status of your request in My Requests.
Oops! Something went wrong.
Looks like we were not able to place your request. Kindly try again later.
Journal Article
Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD
2011
Overview
Duchenne muscular dystrophy (DMD) is a genetic disease affecting about one in every 3,500 boys. This X-linked pathology is due to the absence of dystrophin in muscle fibers. This lack of dystrophin leads to the progressive muscle degeneration that is often responsible for the death of the DMD patients during the third decade of their life. There are currently no curative treatments for this disease but different therapeutic approaches are being studied. Gene therapy consists of introducing a transgene coding for full-length or a truncated version of dystrophin complementary DNA (cDNA) in muscles, whereas pharmaceutical therapy includes the use of chemical/biochemical substances to restore dystrophin expression or alleviate the DMD phenotype. Over the past years, many potential drugs were explored. This led to several clinical trials for gentamicin and ataluren (PTC124) allowing stop codon read-through. An alternative approach is to induce the expression of an internally deleted, partially functional dystrophin protein through exon skipping. The vectors and the methods used in gene therapy have been continually improving in order to obtain greater encapsidation capacity and better transduction efficiency. The most promising experimental approaches using pharmaceutical and gene therapies are reviewed in this article.
Publisher
Elsevier Inc,Elsevier Limited,Nature Publishing Group
Subject
/ Gentamicins - therapeutic use
/ Humans
/ Male
/ Muscle Fibers, Skeletal - chemistry
/ Muscle Fibers, Skeletal - metabolism
/ Muscle Fibers, Skeletal - pathology
/ Muscular Dystrophy, Duchenne - drug therapy
/ Muscular Dystrophy, Duchenne - genetics
/ Muscular Dystrophy, Duchenne - physiopathology
/ Muscular Dystrophy, Duchenne - therapy
/ Mutation
/ Oxadiazoles - therapeutic use
/ Proteins
/ Review
/ Steroids
