Rodino-Klapac, Louise: search on 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:

Year:

2009

Language:

english

File:

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

Year:

2017

Language:

english

File:

PDF, 338 KB

english, 2017

3

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

Year:

2017

Language:

english

File:

PDF, 3.04 MB

english, 2017

4

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

Year:

2010

Language:

english

File:

PDF, 266 KB

english, 2010

5

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:

Year:

2013

Language:

english

File:

PDF, 3.30 MB

english, 2013

6

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

Rodino-Klapac, Louise R.

Year:

2015

Language:

english

File:

PDF, 4.43 MB

english, 2015

7

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

Year:

2014

Language:

english

File:

PDF, 966 KB

english, 2014

8

Clinical Trials of Exon skipping in Duchenne Muscular Dystrophy

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

Journal:

Expert Opinion on Orphan Drugs

Year:

2017

Language:

english

File:

PDF, 1.74 MB

english, 2017

9

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

Year:

2009

Language:

english

File:

PDF, 1.22 MB

english, 2009

10

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

Year:

2010

Language:

english

File:

PDF, 815 KB

english, 2010

11

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

Year:

2016

Language:

english

File:

PDF, 268 KB

english, 2016

12

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

Year:

2011

Language:

english

File:

PDF, 1.43 MB

english, 2011

13

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

Year:

2010

Language:

english

File:

PDF, 1.72 MB

english, 2010

14

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

Year:

2009

Language:

english

File:

PDF, 1.83 MB

english, 2009

15

Emerging drugs for Duchenne muscular dystrophy

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

Journal:

Expert Opinion on Emerging Drugs

Year:

2012

Language:

english

File:

PDF, 512 KB

english, 2012

16

Gene Therapy for Muscular Dystrophy: Moving the Field Forward

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

Journal:

Pediatric Neurology

Year:

2014

Language:

english

File:

PDF, 2.90 MB

english, 2014

17

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

Year:

2010

Language:

english

File:

PDF, 1.42 MB

english, 2010

18

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

Year:

2014

Language:

english

File:

PDF, 1.31 MB

english, 2014

19

AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones

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

Journal:

European Journal of Paediatric Neurology

Year:

2017

Language:

english

File:

PDF, 169 KB

english, 2017

20

Pre-clinical Systemic Delivery of Adeno-Associated Alpha-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy

Griffin, Danielle A, Pozsgai, Eric R, Heller, Kristin N, Potter, Rachael A, Peterson, Ellyn L., Rodino-Klapac, Louise R

Journal:

Human Gene Therapy

Year:

2020

File:

PDF, 1.32 MB

2020

21

Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice

Pozsgai, Eric R., Griffin, Danielle A., Heller, Kristin N., Mendell, Jerry R., Rodino-Klapac, Louise R.

Journal:

Molecular Therapy

Year:

2017

Language:

english

File:

PDF, 4.06 MB

english, 2017

22

480. Gene Therapy for Spinal Muscular Atrophy Type 1 Shows Potential to Improve Survival and Motor Functional Outcomes

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

Journal:

Molecular Therapy

Year:

2016

Language:

english

File:

PDF, 494 KB

english, 2016

23

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

Year:

2013

Language:

english

File:

PDF, 624 KB

english, 2013

24

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

Year:

2016

Language:

english

File:

PDF, 1.05 MB

english, 2016

25

Clinical development on the frontier: gene therapy for duchenne muscular dystrophy

Asher, Damon R., Thapa, Khampaseuth, Dharia, Sachi D., Khan, Navid, Potter, Rachael A., Rodino-Klapac, Louise R., Mendell, Jerry R.

Journal:

Expert Opinion on Biological Therapy

Year:

2020

File:

PDF, 2.45 MB

2020

26

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

Year:

2012

Language:

english

File:

PDF, 1.10 MB

english, 2012

27

622. Systemic β-Sarcoglycan Gene Therapy for Treatment of Cardiac and Skeletal Muscle Deficits in LGMD2E

Pozsgai, Eric, Griffin, Danielle, Heller, Kristin, Mendell, Jerry, Rodino-Klapac, Louise

Journal:

Molecular Therapy

Year:

2016

Language:

english

File:

PDF, 371 KB

english, 2016

28

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

Year:

2013

Language:

english

File:

PDF, 683 KB

english, 2013

29

C-2. Eteplirsen, a Phosphorodiamidate Morpholino Oligomer (PMO) for the Treatment of Duchenne Muscular Dystrophy (DMD): 168 Week Update on Six-Minute Walk Test (6MWT), Pulmonary Function Testing (PFT), and Safety

Mendell, Jerry R., Rodino-Klapac, Louise, Sahenk, Zarife, Rouch, Kandice, Bird, Lauren, Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Lewis, Sarah, Shontz, Kim, Flanigan, Kevin, Shilling, Christoph

Journal:

Molecular Therapy

Year:

2015

Language:

english

File:

PDF, 329 KB

english, 2015

30

Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes

Mendell, Jerry R., Sahenk, Zarife, Al-Zaidy, Samiah, Rodino-Klapac, Louise R., Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Miller, Natalie, Yalvac, Mehmet, Dvorchik, Igor, Moore-Clingenpeel

Journal:

Molecular Therapy

Year:

2017

Language:

english

File:

PDF, 1.27 MB

english, 2017

31

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

Year:

2007

Language:

english

File:

PDF, 1.23 MB

english, 2007

32

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

Year:

2015

Language:

english

File:

PDF, 676 KB

english, 2015

33

Gene Therapy for Duchenne Muscular Dystrophy

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

Journal:

Archives of Neurology

Year:

2007

Language:

english

File:

PDF, 303 KB

english, 2007

34

505. Treatment of DMD 5’ Mutations Through Two Different exon2 Skipping Strategies: Intramuscular Delivery of rAAV9.snRNA Mediated Skipping and Antisense Morpholino Oligomers

Simmons, Tabatha R., Wein, Nicolas, Vulin-Chaffiol, Adeline, Heller, Kristin, Rutherford, Andrea, Rodino-Klapac, Louise, Flanigan, Kevin M.

Journal:

Molecular Therapy

Year:

2015

Language:

english

File:

PDF, 483 KB

english, 2015

35

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

Year:

2015

Language:

english

File:

PDF, 534 KB

english, 2015

36

506. β-Sarcoglycan Gene Transfer Prevents Muscle Fibrosis and Inflammation in an Aged LGMD2E Mouse Model

Pozsgai, Eric R., Griffin, Danielle A., Heller, Kristin N., Mendell, Jerry R., Rodino-Klapac, Louise R.

Journal:

Molecular Therapy

Year:

2015

Language:

english

File:

PDF, 602 KB

english, 2015

37

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

Year:

2016

Language:

english

File:

PDF, 364 KB

english, 2016

38

497. Follistatin Gene Therapy Improves Six Minute Walk Distance in Sporadic Inclusion Body Myositis (sIBM)

Mendell, Jerry R., Sahenk, Zarife, Hogan, Mark, Al-Zaidy, Samiah, Flanigan, Kevin, Rodino-Klapac, Louise R., McColly, Markus, Church, Kathleen, Lewis, Sarah, Lowes, Linda, Alfano, Lindsay, Berry, Kath

Journal:

Molecular Therapy

Year:

2016

Language:

english

File:

PDF, 1.02 MB

english, 2016

39

Induction of T-Cell Infiltration and Programmed Death Ligand 2 Expression by Adeno-Associated Virus in Rhesus Macaque Skeletal Muscle and Modulation by Prednisone

Cramer, Megan L., Shao, Guohong, Rodino-Klapac, Louise R., Chicoine, Louis G., Martin, Paul T.

Journal:

Human Gene Therapy

Year:

2017

Language:

english

File:

PDF, 1.24 MB

english, 2017

40

AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Interim data demonstrates improvements in supportive care use

Shell, Richard, Al-Zaidy, Samiah, Arnold, W. Dave, Rodino-Klapac, Louise, Prior, Thomas W., Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T., Nagendran, Sukumar, L'It

Journal:

European Journal of Paediatric Neurology

Year:

2017

Language:

english

File:

PDF, 43 KB

english, 2017

41

Zebrafish topped is required for ventral motor axon guidance

Louise R. Rodino-Klapac, Christine E. Beattie

Journal:

Developmental Biology

Year:

2004

Language:

english

File:

PDF, 592 KB

english, 2004

42

Update on the Treatment of Duchenne Muscular Dystrophy

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

Journal:

Current Neurology and Neuroscience Reports

Year:

2013

Language:

english

File:

PDF, 206 KB

english, 2013

43

CRISPR/Cas9 treatment for Duchenne muscular dystrophy

Mendell, Jerry R, Rodino-Klapac, Louise R

Journal:

Year:

2016

Language:

english

File:

PDF, 585 KB

english, 2016

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