Rodino-Klapac, L. : rechercher sur Z-Library

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1

Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease

Louise R. Rodino-Klapac, Amanda M. Haidet, Janaiah Kota, Chalonda Handy, Brian K. Kaspar, Jerry R. Mendell

Journal:

Année:

2009

Langue:

english

Fichier:

PDF, 932 KB

english, 2009

2

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Mendell, Jerry R., Al-Zaidy, Samiah, Shell, Richard, Arnold, W. Dave, Rodino-Klapac, Louise R., Prior, Thomas W., Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T., Na

Journal:

New England Journal of Medicine

Année:

2017

Langue:

english

Fichier:

PDF, 338 KB

english, 2017

3

Gentamicin-induced readthrough of stop codons in duchenne muscular dystrophy

Vinod Malik, Louise R. Rodino-Klapac, Laurence Viollet, Cheryl Wall, Wendy King, Roula Al-Dahhak, Sarah Lewis, Christopher J. Shilling, Janaiah Kota, Carmen Serrano-Munuera, John Hayes, John D. Mahan,

Journal:

Annals of Neurology

Année:

2010

Langue:

english

Fichier:

PDF, 266 KB

english, 2010

4

Impaired regeneration in LGMD2A supported by increased PAX7-positive satellite cell content and muscle-specific microrna dysregulation

Rosales, Xiomara Q., Malik, Vinod, Sneh, Amita, Chen, Lei, Lewis, Sarah, Kota, Janaiah, Gastier-Foster, Julie M., Astbury, Caroline, Pyatt, Rob, Reshmi, Shalini, Rodino-Klapac, Louise R., Clark, K. Re

Journal:

Année:

2013

Langue:

english

Fichier:

PDF, 3.30 MB

english, 2013

5

Neuromuscular Disorders of Infancy, Childhood, and Adolescence || Principles and Practice of Molecular Therapies

Rodino-Klapac, Louise R.

Année:

2015

Langue:

english

Fichier:

PDF, 4.43 MB

english, 2015

6

Generation of a new, inducible model of FSHD that develops overt myopathic phenotypes

Giesige, C., Heller, K., Wallace, L., Domire, J., Eidahl, J., Mukweyi, D., Garwick-Coppens, S., Guckes, S., Rodino-Klapac, L., Harper, S.

Journal:

Neuromuscular Disorders

Année:

2017

Langue:

english

Fichier:

PDF, 227 KB

english, 2017

7

Pre-clinical safety and off-target studies to support translation of AAV-mediated RNAi therapy for FSHD

Wallace, Lindsay M., Saad, Nizar Y., Pyne, Nettie K., Fowler, Allison M., Eidahl, Jocelyn O., Domire, Jacqueline S., Griffin, Danielle, Herman, Adam C., Sahenk, Zarife, Rodino-Klapac, Louise, Harper,

Journal:

Molecular Therapy — Methods & Clinical Development

Année:

2017

Langue:

english

Fichier:

PDF, 3.04 MB

english, 2017

8

Limb-girdle muscular dystrophy type 2D gene therapy restores α-sarcoglycan and associated proteins

Jerry R. Mendell, Louise R. Rodino-Klapac, Xiomara Rosales-Quintero, Janaiah Kota, Brian D. Coley, Gloria Galloway, Josepha M. Craenen, Sarah Lewis, Vinod Malik, Christopher Shilling, Barry J. Byrne,

Journal:

Annals of Neurology

Année:

2009

Langue:

english

Fichier:

PDF, 1.22 MB

english, 2009

9

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D

Jerry R. Mendell, Louise R. Rodino-Klapac, Xiomara Q. Rosales, Brian D. Coley, Gloria Galloway, Sarah Lewis, Vinod Malik, Chris Shilling, Barry J. Byrne, Thomas Conlon, Katherine J. Campbell, William

Journal:

Annals of Neurology

Année:

2010

Langue:

english

Fichier:

PDF, 1.72 MB

english, 2010

10

mdx5cv Mice Manifest More Severe Muscle Dysfunction and Diaphragm Force Deficits than Do mdx Mice

Nicholas Beastrom, Haiyan Lu, Allison Macke, Benjamin D. Canan, Eric K. Johnson, Christopher M. Penton, Brian K. Kaspar, Louise R. Rodino-Klapac, Lan Zhou, Paul M.L. Janssen, Federica Montanaro

Journal:

The American Journal of Pathology

Année:

2011

Langue:

english

Fichier:

PDF, 1.43 MB

english, 2011

11

Zebrafish topped is required for ventral motor axon guidance

Louise R. Rodino-Klapac, Christine E. Beattie

Journal:

Developmental Biology

Année:

2004

Langue:

english

Fichier:

PDF, 592 KB

english, 2004

12

Semaphorin 5A is a bifunctional axon guidance cue for axial motoneurons in vivo

Jona D. Hilario, Louise R. Rodino-Klapac, Chunping Wang, Christine E. Beattie

Journal:

Developmental Biology

Année:

2009

Langue:

english

Fichier:

PDF, 1.83 MB

english, 2009

13

Gene therapy for muscular dystrophy: Lessons learned and path forward

Jerry R. Mendell, Louise Rodino-Klapac, Zarife Sahenk, Vinod Malik, Brian K. Kaspar, Christopher M. Walker, K. Reed Clark

Journal:

Neuroscience Letters

Année:

2012

Langue:

english

Fichier:

PDF, 1.10 MB

english, 2012

14

Update on the Treatment of Duchenne Muscular Dystrophy

Rodino-Klapac, Louise R., Mendell, Jerry R., Sahenk, Zarife

Journal:

Current Neurology and Neuroscience Reports

Année:

2013

Langue:

english

Fichier:

PDF, 206 KB

english, 2013

15

A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy

Louise R Rodino-Klapac, Paul ML Janssen, Chrystal L Montgomery, Brian D Coley, Louis G Chicoine, K Reed Clark, Jerry R Mendell

Journal:

Journal of Translational Medicine

Année:

2007

Langue:

english

Fichier:

PDF, 1.23 MB

english, 2007

16

P.11.20 Results at 2years of a phase IIb extension study of the exon-skipping drug eteplirsen in patients with DMD

Mendell, J.R., Rodino-Klapac, L., Sahenk, Z., Roush, K., Bird, L., Lowes, L.P., Alfano, L., Gomez, A.M., Lewis, S., Malik, V., Shontz, K., Flanigan, K.M., Kean, J.R., Allen, H.D., Shilling, C., Sazani

Journal:

Neuromuscular Disorders

Année:

2013

Langue:

english

Fichier:

PDF, 61 KB

english, 2013

17

Gene Therapy for Muscular Dystrophy: Moving the Field Forward

Al-Zaidy, Samiah, Rodino-Klapac, Louise, Mendell, Jerry R.

Journal:

Pediatric Neurology

Année:

2014

Langue:

english

Fichier:

PDF, 2.90 MB

english, 2014

18

G.P.289

Griffin, D., Pozsgai, E., Johnson, R., Grose, W., Heller, K., Mendell, J.R., Sahenk, Z., Rodino-Klapac, L.R.

Journal:

Neuromuscular Disorders

Année:

2014

Langue:

english

Fichier:

PDF, 46 KB

english, 2014

19

G.P.110

Sazani, P., Magee, T., Charleston, J.S., Shanks, C., Zhang, J., Carver, M., Rodino-Klapac, L., Sahenk, Z., Roush, K., Bird, L., Lowes, L.P., Alfano, L., Gomez, A.M., Lewis, S., Malik, V., Shontz, K.,

Journal:

Neuromuscular Disorders

Année:

2014

Langue:

english

Fichier:

PDF, 47 KB

english, 2014

20

G.O.9

Sondergaard, P.C., Griffin, D.A., Pozsgai, E.R., Johnson, R.W., Mendell, J.R., Rodino-Klapac, L.R.

Journal:

Neuromuscular Disorders

Année:

2014

Langue:

english

Fichier:

PDF, 61 KB

english, 2014

21

G.O.24

Mendell, J.R., Rodino-Klapac, L., Sahenk, Z., Roush, K., Bird, L., Lowes, L.P., Alfano, L., Gomez, A.M., Lewis, S., Malik, V., Shontz, K., Flanigan, K.M., Shilling, C., Sazani, P., Saoud, J., Duda, P.

Journal:

Neuromuscular Disorders

Année:

2014

Langue:

english

Fichier:

PDF, 50 KB

english, 2014

22

G.P.227

Pozsgai, E.R., Griffin, D.A., Heller, K.N., Mendell, J.R., Rodino-Klapac, L.R.

Journal:

Neuromuscular Disorders

Année:

2014

Langue:

english

Fichier:

PDF, 46 KB

english, 2014

23

G.O.25

Mendell, J.R., Sahenk, Z., Rodino-Klapac, L.R., Clark, K.R., Lewis, K.R., Shontz, K., Al-Zaidy, S., Alfano, L., Lowes,, L.P., Berry, K., Malik, V., Shilling, C.J., Rosales, X.Q., Walker, C.M., Flaniga

Journal:

Neuromuscular Disorders

Année:

2014

Langue:

english

Fichier:

PDF, 50 KB

english, 2014

24

G.O.26

Heller, K.N., Montgomery, C.L., Shontz, K.M., Clark, K.R., Mendell, J.R., Rodino-Klapac, L.R.

Journal:

Neuromuscular Disorders

Année:

2014

Langue:

english

Fichier:

PDF, 49 KB

english, 2014

25

Eteplirsen for the treatment of Duchenne muscular dystrophy

Mendell, Jerry R., Rodino-Klapac, Louise R., Sahenk, Zarife, Roush, Kandice, Bird, Loren, Lowes, Linda P., Alfano, Lindsay, Gomez, Ann Maria, Lewis, Sarah, Kota, Janaiah, Malik, Vinod, Shontz, Kim, Wa

Journal:

Annals of Neurology

Année:

2013

Langue:

english

Fichier:

PDF, 624 KB

english, 2013

26

Emerging drugs for Duchenne muscular dystrophy

Malik, Vinod, Rodino-Klapac, Louise R., Mendell, Jerry R

Journal:

Expert Opinion on Emerging Drugs

Année:

2012

Langue:

english

Fichier:

PDF, 512 KB

english, 2012

27

Vascular Delivery of rAAVrh74.MCK.GALGT2 to the Gastrocnemius Muscle of the Rhesus Macaque Stimulates the Expression of Dystrophin and Laminin α2 Surrogates

Chicoine, Louis G., Rodino-Klapac, Louise R., Shao, Guohong, Xu, Rui, Bremer, William G., Camboni, Marybeth, Golden, Bethannie, Montgomery, Chrystal L., Shontz, Kimberly, Heller, Kristin N., Griffin,

Journal:

Molecular Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 1.31 MB

english, 2014

28

Persistent Expression of FLAG-tagged Micro dystrophin in Nonhuman Primates Following Intramuscular and Vascular Delivery

Rodino-Klapac, Louise R, Montgomery, Chrystal L, Bremer, William G, Shontz, Kimberly M, Malik, Vinod, Davis, Nancy, Sprinkle, Spencer, Campbell, Katherine J, Sahenk, Zarife, Clark, K Reed, Walker, Chr

Journal:

Molecular Therapy

Année:

2010

Langue:

english

Fichier:

PDF, 1.42 MB

english, 2010

29

AAV1.NT-3 Gene Therapy for Charcot–Marie–Tooth Neuropathy

Sahenk, Zarife, Galloway, Gloria, Clark, Kelly Reed, Malik, Vinod, Rodino-Klapac, Louise R, Kaspar, Brian K., Chen, Lei, Braganza, Cilwyn, Montgomery, Chrystal, Mendell, Jerry R

Journal:

Molecular Therapy

Année:

2014

Langue:

english

Fichier:

PDF, 966 KB

english, 2014

30

Follistatin Gene Delivery Enhances Muscle Growth and Strength in Nonhuman Primates

Kota, J., Handy, C. R., Haidet, A. M., Montgomery, C. L., Eagle, A., Rodino-Klapac, L. R., Tucker, D., Shilling, C. J., Therlfall, W. R., Walker, C. M., Weisbrode, S. E., Janssen, P. M. L., Clark, K.

Journal:

Science Translational Medicine

Année:

2009

Langue:

english

Fichier:

PDF, 1.08 MB

english, 2009

31

MicroRNA Based Treatment of Cardiomyopathy: Not all Dystrophies are Created Equal

Rodino-Klapac, L. R.

Journal:

Journal of the American Heart Association

Année:

2013

Langue:

english

Fichier:

PDF, 878 KB

english, 2013

32

Molecular Therapeutic Strategies Targeting Duchenne Muscular Dystrophy

Mendell, J. R., Rodino-Klapac, L. R., Malik, V.

Journal:

Journal of Child Neurology

Année:

2010

Langue:

english

Fichier:

PDF, 96 KB

english, 2010

33

Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model

Rodino-Klapac, L. R., Janssen, P. M. L., Shontz, K. M., Canan, B., Montgomery, C. L., Griffin, D., Heller, K., Schmelzer, L., Handy, C., Clark, K. R., Sahenk, Z., Mendell, J. R., Kaspar, B. K.

Journal:

Human Molecular Genetics

Année:

2013

Langue:

english

Fichier:

PDF, 1.37 MB

english, 2013

34

Gene Therapy for Duchenne Muscular Dystrophy

Rodino-Klapac, Louise R., Chicoine, Louis G., Kaspar, Brian K., Mendell, Jerry R.

Journal:

Archives of Neurology

Année:

2007

Langue:

english

Fichier:

PDF, 303 KB

english, 2007

35

Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice

Martin, P. T., Xu, R., Rodino-Klapac, L. R., Oglesbay, E., Camboni, M., Montgomery, C. L., Shontz, K., Chicoine, L. G., Clark, K. R., Sahenk, Z., Mendell, J. R., Janssen, P. M. L.

Journal:

AJP Cell Physiology

Année:

2008

Langue:

english

Fichier:

PDF, 919 KB

english, 2008

36

Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D

Rodino-Klapac, L. R., Lee, J-S, Mulligan, R. C., Clark, K. R., Mendell, J. R.

Journal:

Année:

2008

Langue:

english

Fichier:

PDF, 707 KB

english, 2008

37

Human α7 integrin gene (ITGΑ7) delivered by adeno-associated virus extends survival of severely affected dystrophin/utrophin deficient mice

Heller, Kristin N, Montgomery, Chrystal L, Shontz, Kimberly M, Clark, K Reed, Mendell, Jerry R., Rodino-Klapac, Louise R.

Journal:

Human Gene Therapy

Année:

2015

Langue:

english

Fichier:

PDF, 676 KB

english, 2015

38

PP09.16 – 2691: Eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne muscular dystrophy (DMD): 3.2 year update on six-minute walk test (6MWT), pulmonary function testing (PFT), and safety

Kaye, E.M., Mendell, J.R., Rodino-Klapac, L., Sahenk, Z., Roush, K., Lowes, L.P., Alfano, L., Gomez, A.M., Malik, V., Flanigan, K.M., Shilling, C., Duda, P., Saoud, J.

Journal:

European Journal of Paediatric Neurology

Année:

2015

Langue:

english

Fichier:

PDF, 91 KB

english, 2015

39

Dystrophin Immunity in Duchenne's Muscular Dystrophy

Mendell, Jerry R., Campbell, Katherine, Rodino-Klapac, Louise, Sahenk, Zarife, Shilling, Chris, Lewis, Sarah, Bowles, Dawn, Gray, Steven, Li, Chengwen, Galloway, Gloria, Malik, Vinod, Coley, Brian, Cl

Journal:

Boston Medical and Surgical Journal

Année:

2010

Langue:

english

Fichier:

PDF, 815 KB

english, 2010

40

β-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice

E. R. Pozsgai, D. A. Griffin, K. N. Heller, J. R. Mendell, L. R. Rodino-klapac

Année:

2015

Langue:

english

Fichier:

PDF, 5.03 MB

english, 2015

41

Eteplirsen, a Phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne muscular dystrophy (DMD): Clinical update

Kaye, E., Mendell, J., Rodino-Klapac, L., Sahenk, Z., Lowes, L., Alfano, L., Berry, K., Gomez Ramirez, A., Lewis, S., Flanigan, K., Cripe, L., Al-Zaidy, S., Duda, P., Sazani, P., Saoud, J.

Journal:

Neuromuscular Disorders

Année:

2015

Langue:

english

Fichier:

PDF, 205 KB

english, 2015

42

AAV-mediated Overexpression of Human α7 Integrin Leads to Histological and Functional Improvement in Dystrophic Mice

Heller, Kristin N, Montgomery, Chrystal L, Janssen, Paul ML, Clark, K Reed, Mendell, Jerry R, Rodino-Klapac, Louise R

Journal:

Molecular Therapy

Année:

2013

Langue:

english

Fichier:

PDF, 683 KB

english, 2013

43

Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy

Malik, V., Rodino-Klapac, L. R., Viollet, L., Mendell, J. R.

Journal:

Therapeutic Advances in Neurological Disorders

Année:

2010

Langue:

english

Fichier:

PDF, 423 KB

english, 2010

44

Defective membrane fusion and repair in Anoctamin5 -deficient muscular dystrophy

Griffin, Danielle A., Johnson, Ryan W., Whitlock, Jarred M., Pozsgai, Eric R., Heller, Kristin N., Grose, William E., Arnold, W. David, Sahenk, Zarife, Hartzell, H. Criss, Rodino-Klapac, Louise R.

Journal:

Human Molecular Genetics

Année:

2016

Langue:

english

Fichier:

PDF, 1.05 MB

english, 2016

45

CRISPR/Cas9 treatment for Duchenne muscular dystrophy

Mendell, Jerry R, Rodino-Klapac, Louise R

Journal:

Année:

2016

Langue:

english

Fichier:

PDF, 585 KB

english, 2016

46

Eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) for Duchenne muscular dystrophy (DMD): Longitudinal comparison to external controls on six-minute walk test (6MWT) and loss of ambulation (LOA)

Mendell, J., Goemans, N., Rodino-Klapac, L., Sahenk, Z., Lowes, L., Alfano, L., Berry, K., Peterson, E., Lewis, S., Shontz, K., Shao, J., Duda, P., Mercuri, E.

Journal:

Neuromuscular Disorders

Année:

2016

Langue:

english

Fichier:

PDF, 195 KB

english, 2016

47

Long-term treatment with eteplirsen promotes exon 51 skipping and novel dystrophin protein production in Duchenne muscular dystrophy patients

Charleston, J., Schnell, F., Dworzak, J., Donoghue, C., Lewis, S., Rodino-Klapac, L., Sahenk, Z., Shanks, C., Voss, J., DeAlwis, U., Frank, D., Wentworth, B., Mendell, J.

Journal:

Neuromuscular Disorders

Année:

2016

Langue:

english

Fichier:

PDF, 194 KB

english, 2016

48

Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy

Al-Zaidy, Samiah A., Sahenk, Zarife, Rodino-Klapac, Louise R., Kaspar, Brian, Mendell, Jerry R.

Journal:

Journal of Neuromuscular Diseases

Année:

2015

Langue:

english

Fichier:

PDF, 534 KB

english, 2015

49

386. Toxicology for DUX4-Targeted MicroRNAs

Wallace, Lindsay M., Domire, Jacqueline S., Griffin, Danielle A., Rodino-Klapac, Louise R., Harper, Scott Q.

Journal:

Molecular Therapy

Année:

2016

Langue:

english

Fichier:

PDF, 268 KB

english, 2016

50

379. MicroRNA-29 and Micro-Dystrophin Combinatorial Therapy Suppresses Fibrosis and Restores Function to mdx/utrn+/− Mice

Heller, Kristin, Meadows, Eric, Lewis, Sarah, Mendell, Josh, Mendell, Jerry, Rodino-Klapac, Louise

Journal:

Molecular Therapy

Année:

2016

Langue:

english

Fichier:

PDF, 364 KB

english, 2016