Functional rescue of the sarcoglycan complex in the BIO 14.6 hamster using delta-sarcoglycan gene transfer

Molecular Cell
K H HoltK P Campbell

Abstract

Four types of limb-girdle muscular dystrophy (LGMD) are known to be caused by mutations in distinct sarcoglycan genes. The BIO 14.6 hamster is a model for sarcoglycan-deficient LGMD with a deletion in the delta-sarcoglycan (delta-SG) gene. We investigated the function of the sarcoglycan complex and the feasibility of sarcoglycan gene transfer for LGMD using a recombinant delta-SG adenovirus in the BIO 14.6 hamster. We demonstrate extensive long-term expression of delta-sarcoglycan and rescue of the entire sarcoglycan complex, as well as restored stable association of alpha-dystroglycan with the sarcolemma. Importantly, muscle fibers expressing delta-sarcoglycan lack morphological markers of muscular dystrophy and exhibit restored plasma membrane integrity. In summary, the sarcoglycan complex is requisite for the maintenance of sarcolemmal integrity, and primary mutations in individual sarcoglycan components can be corrected in vivo.

References

Sep 20, 1991·Cell·J M Ervasti, K P Campbell
Apr 1, 1973·Virology·F L Graham, A J van der Eb
Jan 1, 1995·Human Molecular Genetics·E OzawaS Noguchi
Apr 1, 1997·Human Molecular Genetics·V NigroG A Puca
Apr 7, 1997·The Journal of Cell Biology·P L McNeil, R A Steinhardt
Apr 1, 1997·Current Opinion in Neurology·V Straub, K P Campbell
Oct 23, 1997·The Journal of Cell Biology·V StraubK P Campbell
Feb 12, 1998·The Journal of Biological Chemistry·R H CrosbieK P Campbell

❮ Previous
Next ❯

Citations

Sep 26, 2000·Muscle & Nerve·R D Cohn, K P Campbell
Sep 14, 2006·Journal of Human Genetics·Motoi Kanagawa, Tatsushi Toda
Nov 20, 2003·Brain Research. Molecular Brain Research·Jianfeng Xiao, Mark S LeDoux
Nov 18, 1998·Current Opinion in Cell Biology·M DurbeejK P Campbell
Oct 6, 1999·Current Opinion in Cell Biology·M D Henry, K P Campbell
Dec 18, 2003·Neuromuscular Disorders : NMD·Kiichiro MatsumuraTeruo Shimizu
Feb 10, 2012·Gene Therapy·Y Lai, D Duan
Dec 14, 2006·Molecular Therapy : the Journal of the American Society of Gene Therapy·Francoise FougerousseIsabelle Richard
Feb 15, 2011·Journal of Biomedicine & Biotechnology·Thomas G HamptonCase G VanDongen
Jul 30, 2011·Skeletal Muscle·Alison M Blain, Volker W Straub
Aug 21, 2013·PloS One·Eric K JohnsonFederica Montanaro
Oct 18, 2001·BioDrugs : Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy·S Takeda, Y Miyagoe-Suzuki
Jul 10, 2003·Proceedings of the National Academy of Sciences of the United States of America·Madeleine DurbeejKevin P Campbell
Jan 27, 2016·Neuromuscular Disorders : NMD·Volker Straub, Marta Bertoli
Mar 22, 2008·The Journal of Urology·Salvatore ArenaVincenzo Di Benedetto
Jan 16, 2007·Experimental Cell Research·Gaynor MillerRachelle H Crosbie
Oct 13, 2011·The Journal of Pathology·Virginie Carmignac, Madeleine Durbeej
Jun 15, 2004·Brain Research. Molecular Brain Research·Akiyo NishiyamaMichihiro Imamura
Jun 21, 2005·Pharmacology & Therapeutics·Tomie KawadaTeruhiko Toyo-Oka
Jul 19, 2013·Biochimica Et Biophysica Acta·Vasco SequeiraJolanda van der Velden
Jun 27, 2012·The Journal of Cell Biology·Jamie L MarshallRachelle H Crosbie-Watson
Dec 29, 1998·The Journal of Cell Biology·Y M ChanL M Kunkel
Apr 6, 1999·The Journal of Cell Biology·R H CrosbieK P Campbell
Sep 23, 1998·The Journal of Cell Biology·F DuclosK P Campbell
Mar 22, 2001·The Journal of Histochemistry and Cytochemistry : Official Journal of the Histochemistry Society·H UedaS Ohno
Feb 19, 2000·Microscopy Research and Technique·S C WatkinsL Billington
May 4, 2017·Journal of Cellular Physiology·Hina F BhatNazir A Ganai
Jun 22, 2018·Current Treatment Options in Neurology·Ava Y Lin, Leo H Wang
Feb 19, 2000·Microscopy Research and Technique·A A HackE M McNally
Apr 28, 1999·FEBS Letters·A SakamotoT Masaki
Feb 23, 2000·FEBS Letters·K H HoltK P Campbell

❮ Previous
Next ❯

Related Concepts

Trending Feeds

COVID-19

Coronaviruses encompass a large family of viruses that cause the common cold as well as more serious diseases, such as the ongoing outbreak of coronavirus disease 2019 (COVID-19; formally known as 2019-nCoV). Coronaviruses can spread from animals to humans; symptoms include fever, cough, shortness of breath, and breathing difficulties; in more severe cases, infection can lead to death. This feed covers recent research on COVID-19.

Blastomycosis

Blastomycosis fungal infections spread through inhaling Blastomyces dermatitidis spores. Discover the latest research on blastomycosis fungal infections here.

Nuclear Pore Complex in ALS/FTD

Alterations in nucleocytoplasmic transport, controlled by the nuclear pore complex, may be involved in the pathomechanism underlying multiple neurodegenerative diseases including Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Here is the latest research on the nuclear pore complex in ALS and FTD.

Applications of Molecular Barcoding

The concept of molecular barcoding is that each original DNA or RNA molecule is attached to a unique sequence barcode. Sequence reads having different barcodes represent different original molecules, while sequence reads having the same barcode are results of PCR duplication from one original molecule. Discover the latest research on molecular barcoding here.

Chronic Fatigue Syndrome

Chronic fatigue syndrome is a disease characterized by unexplained disabling fatigue; the pathology of which is incompletely understood. Discover the latest research on chronic fatigue syndrome here.

Evolution of Pluripotency

Pluripotency refers to the ability of a cell to develop into three primary germ cell layers of the embryo. This feed focuses on the mechanisms that underlie the evolution of pluripotency. Here is the latest research.

Position Effect Variegation

Position Effect Variagation occurs when a gene is inactivated due to its positioning near heterochromatic regions within a chromosome. Discover the latest research on Position Effect Variagation here.

STING Receptor Agonists

Stimulator of IFN genes (STING) are a group of transmembrane proteins that are involved in the induction of type I interferon that is important in the innate immune response. The stimulation of STING has been an active area of research in the treatment of cancer and infectious diseases. Here is the latest research on STING receptor agonists.

Microbicide

Microbicides are products that can be applied to vaginal or rectal mucosal surfaces with the goal of preventing, or at least significantly reducing, the transmission of sexually transmitted infections. Here is the latest research on microbicides.