Laminin {alpha}1 chain reduces muscular dystrophy in laminin {alpha}2 chain deficient mice.
(2004) In Human Molecular Genetics 13(16). p.1775-1784- Abstract
- Laminin (LN) {alpha}2 chain deficiency in humans and mice leads to severe forms of congenital muscular dystrophy (CMD). Here, we investigated whether LN{alpha}1 chain in mice can compensate for the absence of LN{alpha}2 chain and prevent the development of muscular dystrophy. We generated mice expressing a LN{alpha}1 chain transgene in skeletal muscle of LN{alpha}2 chain deficient mice. LN{alpha}1 is not normally expressed in muscle, but the transgenically produced LN{alpha}1 chain was incorporated into muscle basement membranes, and normalized the compensatory changes of expression of certain other laminin chains ({alpha}4, ß2). In 4-month-old mice, LN{alpha}1 chain could fully prevent the development of muscular dystrophy in several... (More)
- Laminin (LN) {alpha}2 chain deficiency in humans and mice leads to severe forms of congenital muscular dystrophy (CMD). Here, we investigated whether LN{alpha}1 chain in mice can compensate for the absence of LN{alpha}2 chain and prevent the development of muscular dystrophy. We generated mice expressing a LN{alpha}1 chain transgene in skeletal muscle of LN{alpha}2 chain deficient mice. LN{alpha}1 is not normally expressed in muscle, but the transgenically produced LN{alpha}1 chain was incorporated into muscle basement membranes, and normalized the compensatory changes of expression of certain other laminin chains ({alpha}4, ß2). In 4-month-old mice, LN{alpha}1 chain could fully prevent the development of muscular dystrophy in several muscles, and partially in others. The LN{alpha}1 chain transgene not only reversed the appearance of histopathological features of the disease to a remarkable degree, but also greatly improved health and longevity of the mice. Correction of LN{alpha}2 chain deficiency by LN{alpha}1 chain may serve as a paradigm for gene therapy of CMD in patients. (Less)
Please use this url to cite or link to this publication:
https://lup.lub.lu.se/record/123960
- author
- Gawlik, Kinga LU ; Miyagoe-Suzuki, Yuko ; Ekblom, Peter LU ; Takeda, Shin'ichi and Durbeej-Hjalt, Madeleine LU
- organization
- publishing date
- 2004
- type
- Contribution to journal
- publication status
- published
- subject
- in
- Human Molecular Genetics
- volume
- 13
- issue
- 16
- pages
- 1775 - 1784
- publisher
- Oxford University Press
- external identifiers
-
- wos:000222880700009
- pmid:15213105
- scopus:4444354572
- ISSN
- 0964-6906
- DOI
- 10.1093/hmg/ddh190
- language
- English
- LU publication?
- yes
- id
- c435c8d8-014f-4035-b2f6-15f78c35ff86 (old id 123960)
- alternative location
- http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=15213105&dopt=Abstract
- date added to LUP
- 2016-04-01 12:10:12
- date last changed
- 2022-01-26 23:47:30
@article{c435c8d8-014f-4035-b2f6-15f78c35ff86, abstract = {{Laminin (LN) {alpha}2 chain deficiency in humans and mice leads to severe forms of congenital muscular dystrophy (CMD). Here, we investigated whether LN{alpha}1 chain in mice can compensate for the absence of LN{alpha}2 chain and prevent the development of muscular dystrophy. We generated mice expressing a LN{alpha}1 chain transgene in skeletal muscle of LN{alpha}2 chain deficient mice. LN{alpha}1 is not normally expressed in muscle, but the transgenically produced LN{alpha}1 chain was incorporated into muscle basement membranes, and normalized the compensatory changes of expression of certain other laminin chains ({alpha}4, ß2). In 4-month-old mice, LN{alpha}1 chain could fully prevent the development of muscular dystrophy in several muscles, and partially in others. The LN{alpha}1 chain transgene not only reversed the appearance of histopathological features of the disease to a remarkable degree, but also greatly improved health and longevity of the mice. Correction of LN{alpha}2 chain deficiency by LN{alpha}1 chain may serve as a paradigm for gene therapy of CMD in patients.}}, author = {{Gawlik, Kinga and Miyagoe-Suzuki, Yuko and Ekblom, Peter and Takeda, Shin'ichi and Durbeej-Hjalt, Madeleine}}, issn = {{0964-6906}}, language = {{eng}}, number = {{16}}, pages = {{1775--1784}}, publisher = {{Oxford University Press}}, series = {{Human Molecular Genetics}}, title = {{Laminin {alpha}1 chain reduces muscular dystrophy in laminin {alpha}2 chain deficient mice.}}, url = {{http://dx.doi.org/10.1093/hmg/ddh190}}, doi = {{10.1093/hmg/ddh190}}, volume = {{13}}, year = {{2004}}, }