Jeffrey S. Chamberlain

Sarcolemma-localized nNOS is required to maintain activity after mild exercise (2008)

Kobayashi, Yvonne M., Rader, Erik P., Crawford, Robert W., Iyengar, Nikhil K., Thedens, Daniel R., Faulkner, John A., ...

Many neuromuscular conditions are characterized by an exaggerated exercise- induced fatigue response that is disproportionate to activity level. This fatigue is not necessarily correlated with...

Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy (2008)

Kimura, En, Han, Jay J., Li, Sheng, Fall, Brent, Ra, Jennifer, Haraguchi, Miki, ...

Duchenne muscular dystrophy (DMD) is characterized in skeletal muscle by cycles of myofiber necrosis and regeneration leading to loss of muscle fibers and replacement with fibrotic connective and...

Molecular and cellular adaptations to chronic myotendinous strain injury in mdx mice expressing a truncated dystrophin (2008)

Banks, Glen B., Combs, Ariana C., Chamberlain, Joel R., Chamberlain, Jeffrey S.

Myotendinous strain injury is the most common injury of human skeletal muscles because the majority of muscle forces are transmitted through this region. Although the immediate response to strain...

Functional capacity of dystrophins carrying deletions in the N-terminal actin-binding domain (2007)

Banks, Glen B., Gregorevic, Paul, Allen, James M., Finn, Eric E., Chamberlain, Jeffrey S.

Duchenne muscular dystrophy and Becker muscular dystrophy (BMD) are caused by mutations in the dystrophin gene. Although many in-frame deletions in the dystrophin gene lead to mild cases of BMD,...

Enhanced effect of microdystrophin gene transfection by HSV-VP22 mediated intercellular protein transport (2007)

Xiong, Fu, Xiao, Shaobo, Yu, Meijuan, Li, Wanyi, Zheng, Hui, Shang, Yanchang, ...

Abstract Background Duchenne musclar dystrophy (DMD) is an X-linked recessive disease caused by mutations of dystrophin gene, there is no effective treatment for this disorder at present....

Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells (2007)

Tonlorenzi, Rossana, Tagliafico, Enrico, Sacchetti, Benedetto, Perani, Laura, ...

Cells derived from blood vessels of human skeletal muscle can regenerate skeletal muscle, similarly to embryonic mesoangioblasts. However, adult cells do not express endothelial markers, but instead...

A highly functional mini-dystrophin/GFP fusion gene for cell and gene therapy studies of Duchenne muscular dystrophy (2006)

Li, Sheng, Kimura, En, Ng, Rainer, Fall, Brent M., Meuse, Leonard, Reyes, Morayma, ...

A promising approach for treating Duchenne muscular dystrophy (DMD) is by autologous cell transplantation of myogenic stem cells transduced with a therapeutic expression cassette. Development of this...

A highly functional mini-dystrophin/GFP fusion gene for cell and gene therapy studies of Duchenne muscular dystrophy (2006)

Li, Sheng, Kimura, En, Ng, Rainer, Fall, Brent M., Meuse, Leonard, Reyes, Morayma, ...

A promising approach for treating Duchenne muscular dystrophy (DMD) is by autologous cell transplantation of myogenic stem cells transduced with a therapeutic expression cassette. Development of this...

Characterization of ARC, apoptosis repressor interacting with CARD, in normal and dystrophin-deficient skeletal muscle (2003)

Abmayr, S., Crawford, R. W., Chamberlain, Jeffrey S.

Duchenne muscular dystrophy (DMD) is an x-linked recessive disorder, primarily characterized by progressive muscle weakness and wasting. The disease results from the absence of dystrophin, however...

Characterization of ARC, apoptosis repressor interacting with CARD, in normal and dystrophin-deficient skeletal muscle (2003)

Abmayr, S., Crawford, R. W., Chamberlain, Jeffrey S.

Duchenne muscular dystrophy (DMD) is an x-linked recessive disorder, primarily characterized by progressive muscle weakness and wasting. The disease results from the absence of dystrophin, however...

Gutted adenoviral vector growth using E1/E2b/E3-deleted helper viruses (2002)

Barjot, Catherine, Hartigan-O'Connor, Dennis, Salvatori, Giovanni, Scott, Jeannine M., Chamberlain, Jeffrey S.

Background Helper-dependent, or gutted, adenoviruses (Ad) lack viral coding sequences, resulting in reduced immunotoxicity compared with conventional Ad vectors. Gutted Ad growth requires a...

Gene therapy of muscular dystrophy (2002)

Chamberlain, Jeffrey S.

Development of gene therapy for the muscular dystrophies represents a daunting challenge requiring significant advances in our knowledge of the defective genes, muscle promoters, viral vectors,...

Spectrin-like repeats from dystrophin and {alpha}-actinin-2 are not functionally interchangeable (2002)

Harper, Scott Q., Crawford, Robert W., DelloRusso, Christiana, Chamberlain, Jeffrey S.

Mutations in the dystrophin gene result in Duchenne muscular dystrophy (DMD). Dystrophin is a multidomain protein that functions to stabilize the sarcolemmal membrane during muscle contraction. The...

Expression of Dp260 in muscle tethers the actin cytoskeleton to the dystrophin-glycoprotein complex and partially prevents dystrophy (2002)

Warner, Laura E., DelloRusso, Christiana, Crawford, Robert W., Rybakova, Inna N., Patel, Jitandrakumar R., Ervasti, James M., ...

Dystrophin forms a mechanical link between the actin cytoskeleton and the extracellular matrix in muscle that helps maintain sarcolemmal integrity. Two regions of dystrophin have been shown to bind...

Tibialis anterior muscles in mdx mice are highly susceptible to contraction-induced injury (2001)

Dellorusso, Christiana, Crawford, Robert W., Chamberlain, Jeffrey S., Brooks, Susan V.

Skeletal muscles of patients with Duchenne muscular dystrophy (DMD) and mdx mice lack dystrophin and are more susceptible to contraction-induced injury than control muscles. Our purpose was to...

Suppression of revertant fibers in mdx mice by expression of a functional dystrophin (2001)

Crawford, Gregory E., Lu, Qi Long, Partridge, Terry A., Chamberlain, Jeffrey S.

Duchenne muscular dystrophy (DMD) is characterized by progressive muscle degeneration that results from the absence of dystrophin. Despite null mutations in the dystrophin gene, many DMD patients...

Dystrophin and utrophin influence fiber type composition and post-synaptic membrane structure (2000)

Rafael, Jill A., Townsend, Elizabeth R., Squire, Sarah E., Potter, Allyson C., Chamberlain, Jeffrey S., Davies, Kay E.

The X-linked muscle wasting disease Duchenne muscular dystrophy is caused by the lack of dystrophin in muscle. Protein structure predictions, patient mutations, in vitro binding studies and...

The mdx-amplification-resistant mutation system assay, a simple and rapid polymerase chain reaction-based detection of the mdx allele (1996)

Amalfitano, Andrea, Chamberlain, Jeffrey S.

The mdx mouse is a murine genetic equivalent of the human X-linked lethal disorder, Duchenne muscular dystrophy (DMD). A number of studies utilizing the mdx mouse have demonstrated the feasibility of...

Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice (1995)

Phelps, Stephanie F., Hauser, Michael A., Cole, Neil M., Rafael, Jill A., Hinkle, Richard T., Faulkner, John A., ...

Duchenne and Becker muscular dystrophy are caused by defects in the dystrophin gene, and are candidates for treatment by gene therapy. We have shown previously that overexpression of a full-length...

Characterization of 10 New Polymorphic Dinucleotide Repeats and Generation of a High-Density Microsatellite-Based Physical Map of the BRCA1 Region of Chromosome 17q21 (1994)

Couch, Fergus J., Kiousis, Sam, Castilla, Lucio H., Xu, Junzhe, Chandrasekharappa, Settara C., Chamberlain, Jeffrey S., ...

A familial early onset breast cancer gene (BRCA1) has been localized to chromosome 17q21. To aid in the identification of this gene a number of new microsatellite markers from the D17S857 to D17S78...

Deletion analysis of the dystrophin-actin binding domain (1994)

Corrado, Kathleen, Mills, P. L., Chamberlain, Jeffrey S.

Three sequence motifs at the N-terminus of dystrophin have previously been proposed to be important for binding to actin. By analyzing a series of purified bacterial fusion proteins deleted for each...

The γ phosphorylase kinase gene, Phkg , maps to mouse Chromosome 5 near Gus (1994)

Chamberlain, Jeffrey S., Maichele, Andrea J.

Phosphorylase kinase is a multimeric regulatory enzyme in the glycogenolytic pathway. Interest in various types of phosphorylase kinase enzyme deficiency has focused attention on cloning and mapping...

Prevention of dystrophic pathology in mdx mice by a truncated dystrophin isoform (1994)

Rafael, Jill A., Sunada, Yoshihide, Cole, Neil M., Campbell, Kevin P., Faulkner, John A., Chamberlain, Jeffrey S.

The C-terminal domain of dystrophin is alternatively spliced to produce a variety of tissue and developmental stage-specific isoforms. Recent studies suggest that the C-terminal domain binds to the...

Phosphoenolpyruvate Carboxykinase (GTP): Characterization of the Human PCK1 Gene and Localization Distal to MODY on Chromosome 20 (1993)

Ting, Chao-Nan, Burgess, Daniel L., Chamberlain, Jeffrey S., Keith, Tim P., Falls, Kathleen, Meisler, Miriam H.

The human PCK1 gene encoding phosphoenolpyruvate carboxykinase (GTP) (PEPCK) was isolated and sequenced. There is 91% amino acid sequence identity (567/622 residues) between the human and the rat...

A B2 Repeat Insertion Generates Alternate Structures of the Mouse Muscle [gamma]-Phosphorylase Kinase Gene (1993)

Maichele, Andrea J., Farwell, Nancy J., Chamberlain, Jeffrey S.

A variety of cDNA and genomic clones for the [gamma]-subunit of mouse muscle phosphorylase kinase (Phk-[gamma]M) have been isolated and characterized. The murine gene for Phk-[gamma]M (Phkg) exhibits...

Identification of new markers in Xp21 between DXS28 (C7) and DMD (1992)

Worley, K. C., Towbin, J. A., Zhu, X. M., Barker, D. F., Ballabio, Andrea, Chamberlain, Jeffrey S., ...

Characterization of Xp21 distal to Duchenne muscular dystrophy (DMD) in the region containing the genes for adrenal hypoplasia congenita (AHC) and glycerol kinase deficiency (GKD) has been limited...

Human and murine dystrophin mRNA transcripts are differentially expressed during skeletal muscle, heart, and brain development (1992)

Bies, Roger D., Phelps, Stephanie F., Cortez, M.Dolores, Roberts, Robert, Caskey, C.Thomas, Chamberlain, Jeffrey S.

Dystrophin transcripts were shown to be alternatively spliced in a pattern characteristic of both tissue type and developmental stage. Multiple novel spliced forms of dystrophin mRNA were identified...

Duchenne muscular dystrophy (1991)

Chamberlain, Jeffrey S.

Progress in understanding the role of dystrophin raises promising hopes for a treatment for Duchenne muscular dystrophy. In addition, great improvements have been made in the ability to diagnose this...

Screening for steroid sulfatase (STS) gene deletions by multiplex DNA amplification (1990)

Caskey, C. Thomas, Ranier, Joel E., Ballabio, Andrea, Chamberlain, Jeffrey S., Zollo, Massimo

Deletions are the most common molecular defect in steroid sulfatase (STS) deficiency. We describe the application of multiplex DNA amplification, by polymerase chain reaction, for deletion screening...

Deletion screening of the Duchenne muscular dystrophy locus via multiplex DNA amplification (1988)

Chamberlain, Jeffrey S., Gibbs, Richard A., Rainer, Joel E., Nguyen, Phi Nga, Thomas, C.

The application of recombinant DNA technology to prenatal diagnosis of many recessively inherited X-linked diseases is complicated by a high frequency of heterogenous, new mutations (1). Partial gene...

Improved Production of Gutted Adenovirus in Cells Expressing Adenovirus Preterminal Protein and DNA Polymerase

Hartigan-O’Connor, Dennis, Amalfitano, Andrea, Chamberlain, Jeffrey S.

Production of gutted, or helper-dependent, adenovirus vectors by current methods is inefficient. Typically, a plasmid form of the gutted genome is transfected with helper viral DNA into 293 cells;...

Production and Characterization of Improved Adenovirus Vectors with the E1, E2b, and E3 Genes Deleted

Amalfitano, Andrea, Hauser, Michael A., Hu, Huimin, Serra, Delila, Begy, Catherine R., Chamberlain, Jeffrey S.

Adenovirus (Ad)-based vectors have great potential for use in the gene therapy of multiple diseases, both genetic and nongenetic. While capable of transducing both dividing and quiescent cells...

Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin

DelloRusso, Christiana, Scott, Jeannine M., Hartigan-O'Connor, Dennis, Salvatori, Giovanni, Barjot, Catherine, Robinson, Ann S., ...

Duchenne muscular dystrophy is a lethal X-linked recessive disorder caused by mutations in the dystrophin gene. Delivery of functionally effective levels of dystrophin to immunocompetent, adult mdx...

Long-term persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation

Gussoni, Emanuela, Bennett, Richard R., Muskiewicz, Kristina R., Meyerrose, Todd, Nolta, Jan A., Gilgoff, Irene, ...

Duchenne muscular dystrophy (DMD) is a severe progressive muscle-wasting disorder caused by mutations in the dystrophin gene. Studies have shown that bone marrow cells transplanted into lethally...

Systemic delivery of genes to striated muscles using adeno-associated viral vectors

Gregorevic, Paul, Blankinship, Michael J, Allen, James M, Crawford, Robert W, Meuse, Leonard, Miller, Daniel G, ...

A major obstacle limiting gene therapy for diseases of the heart and skeletal muscles is an inability to deliver genes systemically to muscles of an adult organism. Systemic gene transfer to striated...

BRCA1 maps proximal to D17S579 on chromosome 17q21 by genetic analysis

Chamberlain, Jeffrey S., Boehnke, Michael, Frank, Thomas S., Kiousis, Sam, Xu, Junxhe, Guo, Sun-Wei, ...

Previous studies have demonstrated linkage between early-onset breast cancer and ovarian cancer and genetic markers on chromosome 17q21. These markers define the location of a gene (BRCA1) which...

Improved Production of Gutted Adenovirus in Cells Expressing Adenovirus Preterminal Protein and DNA Polymerase

Hartigan-O’Connor, Dennis, Amalfitano, Andrea, Chamberlain, Jeffrey S.

Production of gutted, or helper-dependent, adenovirus vectors by current methods is inefficient. Typically, a plasmid form of the gutted genome is transfected with helper viral DNA into 293 cells;...

Production and Characterization of Improved Adenovirus Vectors with the E1, E2b, and E3 Genes Deleted

Amalfitano, Andrea, Hauser, Michael A., Hu, Huimin, Serra, Delila, Begy, Catherine R., Chamberlain, Jeffrey S.

Adenovirus (Ad)-based vectors have great potential for use in the gene therapy of multiple diseases, both genetic and nongenetic. While capable of transducing both dividing and quiescent cells...

Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin

DelloRusso, Christiana, Scott, Jeannine M., Hartigan-O'Connor, Dennis, Salvatori, Giovanni, Barjot, Catherine, Robinson, Ann S., ...

Duchenne muscular dystrophy is a lethal X-linked recessive disorder caused by mutations in the dystrophin gene. Delivery of functionally effective levels of dystrophin to immunocompetent, adult mdx...

Long-term persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation

Gussoni, Emanuela, Bennett, Richard R., Muskiewicz, Kristina R., Meyerrose, Todd, Nolta, Jan A., Gilgoff, Irene, ...

Duchenne muscular dystrophy (DMD) is a severe progressive muscle-wasting disorder caused by mutations in the dystrophin gene. Studies have shown that bone marrow cells transplanted into lethally...

BRCA1 maps proximal to D17S579 on chromosome 17q21 by genetic analysis

Chamberlain, Jeffrey S., Boehnke, Michael, Frank, Thomas S., Kiousis, Sam, Xu, Junxhe, Guo, Sun-Wei, ...

Previous studies have demonstrated linkage between early-onset breast cancer and ovarian cancer and genetic markers on chromosome 17q21. These markers define the location of a gene (BRCA1) which...

Force and power output of fast and slow skeletal muscles from mdx mice 6-28 months old

Lynch, Gordon S, Hinkle, Richard T, Chamberlain, Jeffrey S, Brooks, Susan V, Faulkner, John A

Differences in the effect of age on structure-function relationships of limb muscles of mdx (dystrophin null) and control mice have not been resolved. We tested the hypotheses that, compared with...

Animal Models for Muscular Dystrophy Show Different Patterns of Sarcolemmal Disruption

Straub, Volker, Rafael, Jill A., Chamberlain, Jeffrey S., Campbell, Kevin P.

Genetic defects in a number of components of the dystrophin–glycoprotein complex (DGC) lead to distinct forms of muscular dystrophy. However, little is known about how alterations in the DGC are...

Assembly of the Dystrophin-Associated Protein Complex Does Not Require the Dystrophin Cooh-Terminal Domain

Crawford, Gregory E., Faulkner, John A., Crosbie, Rachelle H., Campbell, Kevin P., Froehner, Stanley C., Chamberlain, Jeffrey S.

Dystrophin is a multidomain protein that links the actin cytoskeleton to laminin in the extracellular matrix through the dystrophin associated protein (DAP) complex. The COOH-terminal domain of...

Functional Deficits in nNOSμ-Deficient Skeletal Muscle: Myopathy in nNOS Knockout Mice

Percival, Justin M., Anderson, Kendra N. E., Gregorevic, Paul, Chamberlain, Jeffrey S., Froehner, Stanley C.

Skeletal muscle nNOSμ (neuronal nitric oxide synthase mu) localizes to the sarcolemma through interaction with the dystrophin-associated glycoprotein (DAG) complex, where it synthesizes nitric oxide...

Biodistribution and Safety Profile of Recombinant Adeno-Associated Virus Serotype 6 Vectors following Intravenous Delivery ▿

Stone, Daniel, Liu, Ying, Li, Zong-Yi, Strauss, Robert, Finn, Eric E., Allen, James M., ...

Recombinant adeno-associated virus vectors based on serotype 6 (rAAV6) efficiently transduce skeletal muscle after intravenous administration and have shown efficacy in the mdx model of muscular...

Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy

Lai, Yi, Thomas, Gail D., Yue, Yongping, Yang, Hsiao T., Li, Dejia, Long, Chun, ...

Sarcolemma-associated neuronal NOS (nNOS) plays a critical role in normal muscle physiology. In Duchenne muscular dystrophy (DMD), the loss of sarcolemmal nNOS leads to functional ischemia and muscle...

Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy

Kimura, En, Han, Jay J., Li, Sheng, Fall, Brent, Ra, Jennifer, Haraguchi, Miki, ...

Duchenne muscular dystrophy (DMD) is characterized in skeletal muscle by cycles of myofiber necrosis and regeneration leading to loss of muscle fibers and replacement with fibrotic connective and...