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Duchenne Muscular Dystrophy: Advances in Therapeutics

by: Chamberlain; Jeffrey S.

Duchenne Muscular Dystrophy: Advances in Therapeutics

by: Chamberlain; Jeffrey S.

ISBN 13:
9780824723255
ISBN 10:
0824723252
Format: Hardcover
Copyright: 02/27/2006
Publisher: CRC Press

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Summary

Guide provides overview of the latest experimental approaches to the treatment of Duchenne Muscular Dystrophy and analyzes clinical genetic, and other aspects of the disease in the context of emerging therapeutic modalities. Emphasizes the importance of acute diagnosis, carrier detection, genetic counseling, and more. For practitioners.

Preface

iii

Contributors

xiii

Introduction

xvii

SECTION I: DUCHENNE MUSCULAR DYSTROPHY BACKGROUND

Clinical Overview of Duchenne Muscular Dystrophy

(20)

Richard T. Moxley III

Historical Background

(3)

Clinical Manifestations

(5)

Diagnosis

(4)

Treatment

(3)

Questions and Opportunities Related to Future Treatment for DMD

(2)

References

(3)

The Functional Biology of Dystrophin: Structural Components and the Pathogenesis of Duchenne Muscular Dystrophy

(34)

Eijiro Ozawa

Keynotes on the Pathogenesis of Duchenne Muscular Dystrophy

(3)

Sarcolemmal and Cytoskeletal Architectures Related to DMD

(9)

Structural Defects in DMD

(5)

Transverse Fixation System: The Structural Basis for a Mechanical Hypothesis of DMD Pathogenesis

(3)

What Induces the Degenerative Processes in DMD?

(5)

Epilogue

(1)

References

(8)

The Functional Biology of Dystrophin: Associated Signaling Pathways and Potential Targets for Therapeutic Intervention

(22)

Gayle Smythe

Steven J. Winder

Thomas A. Rando

Introduction

(2)

Dystrophin Domains and Protein Interactions

(4)

Cellular Signaling Pathways Associated with the DAPC

(6)

Conclusions

(1)

References

(9)

SECTION II: DIAGNOSTIC CONSIDERATIONS

Duchenne Muscular Dystrophy and Becker Muscular Dystrophy: Diagnostic Principles

(14)

H. B. Ginjaar

J. T. den Dunnen

E. Bakker

G. J. B. van Ommen

Introduction

(1)

Confirmation Diagnosis of DMD/BMD

(5)

Prenatal Diagnosis

(1)

Concluding Remarks

(2)

References

(5)

Mutation Detection

(14)

Thomas W. Prior

Gene Studies

(3)

Molecular Diagnostics

(7)

Point Mutation Detection in the Dystrophin Gene

101

(2)

Conclusions

103

(1)

References

103

(2)

Protein Studies in Duchenne Muscular Dystrophy

105

(18)

Kathryn North

Sandra Cooper

Background

105

(7)

Methodological Approach to Protein Diagnosis of Dystrophinopathies

112

(6)

References

118

(5)

SECTION III: CURRENT THERAPEUTICS

Medical Management of Duchenne Muscular Dystrophy

123

(26)

Roula Al-Dahhak

John T. Kissel

Introduction

123

(3)

Corticosteroids

126

(9)

Other Agents

135

(5)

Summary and Conclusions

140

(1)

References

141

(8)

Rehabilitation Management of Duchenne Muscular Dystrophy

149

(28)

Craig M. McDonald

Gregory T. Carter

Jay J. Han

Joshua O. Benditt

Introduction

149

(1)

Initial Rehabilitation Evaluation

150

(4)

Evaluating Disease Progression

154

(1)

Rehabilitation Management

154

(13)

The Role of Exercise

167

(2)

Improving Functional Mobility

169

(1)

References

170

(7)

Orthopedic Interventions in the Management of Duchenne Muscular Dystrophy: A Review of Current Practice and Clinical Outcomes

177

(24)

Irwin M. Siegel

John D. Hsu

Introduction

177

(1)

Biomechanics

177

(2)

Management

179

(1)

Stage I: Diagnosis Until 8 to 9 Years of Age

180

(3)

Fractures

183

(1)

Stage II: 9 to 12 Years of Age

184

(1)

Lower Extremity Surgery

184

(3)

Compartment Syndrome

187

(1)

Talipes Equinovarus

188

(1)

Stage III: Full-Time Sitting

189

(2)

Scoliosis

191

(6)

Summary

197

(1)

References

197

(4)

SECTION IV: EXPERIMENTAL THERAPEUTICS

Therapeutic Principles and Challenges in Duchenne Muscular Dystrophy

201

(26)

Dominic J. Wells

Introduction

201

(1)

Animal Models

202

(3)

Gene Therapeutic Approaches to DMD

205

(6)

Pharmacological Therapeutic Approaches to DMD

211

(3)

Potential Problems in Translating Laboratory Studies into Clinical Treatments

214

(2)

Conclusions

216

(1)

References

217

(10)

Experimental Pharmacologic Therapies in Duchenne Dystrophy: Current Clinical Trials

227

(24)

Shannon L. Venance

Robert C. Griggs

Introduction

227

(5)

Preclinical Studies

232

(1)

Active Clinical Trials

233

(5)

Future Directions

238

(2)

IGF-I

240

(1)

Conclusions

241

(1)

References

241

(10)

Utrophin: The Intersection Between Pharmacological and Genetic Therapy

251

(28)

Kelly J. Perkins

Kay E. Davies

Introduction

251

(1)

The Utrophin Gene and Protein

252

(2)

Animal Models

254

(1)

Promoter Studies of Utrophin

255

(8)

Other Effectors of Utrophin Upregulation

263

(1)

Utrophin-Based Therapeutic Strategies

263

(4)

Conclusions

267

(1)

References

267

(12)

Regenerative Therapy

279

(16)

J. E. Morgan

Karima Brimah

C. A. Collins

T. A. Partridge

Introduction

279

(1)

Hypothetical Mechanisms

280

(1)

Regeneration of Skeletal Muscle from Muscle Precursor Cells

281

(2)

Growth Factors as Regenerative Therapy for Myopathic or Aged Muscles

283

(2)

Stem Cells in Skeletal Muscle

285

(1)

Conclusions

286

(1)

References

287

(8)

Cellular Mediated Delivery: The Intersection Between Regenerative Medicine and Genetic Therapy

295

(24)

K. Liadaki

F. Montanaro

L. M. Kunkel

Introduction

295

(1)

Muscle Cells Available for Therapy of DMD

296

(2)

Delivery Methods

298

(1)

Intramuscular Transplants of Myoblasts

299

(2)

Donor Cell Selection

301

(1)

Muscle Side Population Cells

302

(1)

Other Muscle-Derived Cells Available for Cell-Mediated Therapy of DMD

303

(3)

Nonmuscle-Derived Cells Participating in Muscle Repair

306

(1)

Stem Cells Derived from Dorsal Aorta (Mesangioblasts)

307

(1)

BM-Derived Cells with Myogenic Potential

308

(1)

Mesenchymal Stem Cells

309

(1)

Skin

309

(1)

Conclusions

310

(1)

References

311

(8)

Oligonucleotide-Mediated Exon Skipping and Gene Editing for Duchenne Muscular Dystrophy

319

(24)

Carmen Bertoni

Thomas A. Rando

Introduction

319

(1)

AONs

320

(6)

Oligonucleotide-Mediated Gene Editing

326

(9)

Other Technologies

335

(2)

Conclusions

337

(1)

References

337

(6)

The Intravascular Delivery of Naked DNA for Treating Duchenne Muscular Dystrophy

343

(20)

Jon A. Wolff

Hans Herweijer

Introduction

343

(2)

Plasmid DNA Delivery

345

(3)

Intravascular Delivery of Naked Plasmid DNA

348

(5)

mdx Mouse Studies

353

(5)

Future Prospects

358

(1)

References

359

(4)

Adenoviral-Mediated Gene Therapy

363

(28)

Laura Goldberg

Paula R. Clemens

Introduction

363

(1)

Adenoviral Capsid Structure

364

(1)

Pathway of Infection

365

(3)

Adenoviral Vectors

368

(4)

Immunity Induced by Adenoviral Vectors

372

(3)

Targeting Adenoviral Vectors

375

(3)

Future Challenges

378

(1)

References

379

(12)

Retroviridae-Based Gene Transfer Vectors in Duchenne Muscular Dystrophy Therapy

391

(22)

Michael L. Roberts

George Dickson

Michael Themis

Molecular Biology of Retroviral Vectors and Their Application in DMD

391

(5)

Retroviral-Mediated Gene Delivery to Muscle Cells: In Vitro and Ex Vivo Approaches

396

(2)

Efficacy of Muscle-Directed Retroviral Delivery In Vivo

398

(1)

Cell-Based Strategies of Muscle-Directed Retroviral Delivery

399

(3)

Modern Approaches to Muscle-Directed Retroviral Delivery

402

(3)

The Development of Lentiviral Vectors for DMD Gene Therapy

405

(3)

Future Prospects

408

(1)

References

409

(4)

Gene Therapy of Muscular Dystrophy Using Adeno-Associated Viral Vectors: Promises and Limitations

413

(26)

Michael J. Blankinship

Paul Gregorevic

Jeffrey S. Chamberlain

Overview

413

(1)

AAV: The Genome and Capsid

413

(1)

AAV Serotypes

414

(1)

rAAV Cloning and Production

415

(2)

Safety of rAAV Vectors

417

(2)

Engineering of Microdystrophins

419

(3)

Dystrophin Replacement in Animal Models of DMD

422

(2)

rAAV Delivery of Nondystrophin Genes for the Treatment of DMD

424

(2)

Delivery of rAAV Vectors

426

(2)

Transduction of Myogenic Precursor Cells by rAAV Vectors

428

(1)

Conclusions

429

(1)

References

429

(10)

Regional and Systemic Gene Delivery Using Viral Vectors

439

(14)

Leonard T. Su

Hansell H. Stedman

Introduction

439

(1)

Direct Intramuscular Injection

440

(1)

Regional Delivery

440

(8)

Future Directions: Applications of the Current Technology

448

(1)

References

449

(4)

Index

453

Amazon no longer offers textbook rentals. We do!

Duchenne Muscular Dystrophy: Advances in Therapeutics

Duchenne Muscular Dystrophy: Advances in Therapeutics

9780824723255

0824723252

Summary

Table of Contents

Supplemental Materials

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