Chapter
1. Introduction
1.1. Promising Therapeutic Avenue
1.2. Discovery of RNAi
1.3. RNAi Mechanism
1.4. Gene-Silencing Technology
Aptamers
Ribozymes
Antisense
Difficulties Plaguing Antisense Clinical Trials
1.5. Gene Therapy
Approaches to Gene Therapy
Latest Advances in RNAi Applications to Gene Therapy
Chapter 2. RNAi in Discovery and Development
2.1. Criteria to be Satisfied before Moving into RNAi Clinical Trials
2.2. Targeting and Validation Using RNAi
2.3. RNAi in Clinical Trials for (Wet) Age-Related Macular Degeneration
Acuity Pharmaceuticals
Sirna Therapeutics
Chapter 3. Prospects for Curing Disease By RNAi Gene Silencing
3.1. RNAi in Viral and Infectious Disease States
Human Papilloma Virus
Human Immunodeficiency Virus Type I
Rotavirus
Malaria
Influenza
3.2. RNAi and Diabetes
3.3. RNAi and Cancer
Progress in In Vitro RNAi Work with Cultured Cells
Progress in In Vivo Gene Silencing
3.4. Prospects of Improving Existing Treatment for Chronic Myelogenous Leukemia
Patients by RNAi
3.5. RNAi as a New Kind of Gene Therapy: Therapeutic Potential and Specific
Applications
RNAi and Spinocerebellar Ataxia
RNAi-Mediated Gene Therapy and Intracranial Tumor
Transposon-Delivered RNAi and Huntington’s Disease
Advantages and Challenges of RNAi Therapy
Chapter 4. RNA Delivery
4.1. Challenges to Overcome in Delivering RNA
4.2. Standard Delivery Methods
Electroporation
Viral Delivery
Lipid Delivery
Systemic Delivery
4.3. Is Delivery of Stabilized RNAi Molecules Essential?
Use of Plasmid Vectors
4.4. Detection/Proof of Knockdown Efficiency In Vivo
4.5. Specific Academic and Industry Concerns about RNAi Delivery
Chapter 5. Market and Market Potential
Chapter 6. Profiles of Companies with Primary Interest in RNAi Therapeutics
6.1. Background
6.2. Company Profiles
Acuity Pharmaceuticals
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Alnylam Pharmaceuticals
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Atugen
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Benitec
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Calando Pharmaceuticals
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
CytRx Corporation
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Devgen
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Galenea
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Intradigm Corporation
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Nastech Pharmaceutical Company
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Nucleonics Inc.
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Santaris Pharma
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Sirna Therapeutics
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Chapter 7. Profiles of Companies in Discovery/Research/ Reagents/Services in
the RNAi Market
7.1. Background
7.2. Company Profiles
Ambion
-Activities
-History
-Research and Development/Products
-Business Strategy
Cenix Bioscience
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
CombiMatrix
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
Dharmacon
Activities
-History
-Research and Development/Products
-Business Strategy
Imgenex Corporation
-Activities
-History
-Research and Development/Products
-Business Strategy
Invitrogen
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
InvivoGen
Activities
-History
-Research and Development/Products
-Business Strategy
Mirus Bio
-Activities
-History
-Research and Development/Products
-Business Strategy
Qiagen
-Activities
-History
-Research and Development/Products
-Business Strategy
-Challenges
SomaGenics
-Activities
-History
-Research and Development/Products
-Business Strategy
Chapter 8. Expert Interviews
8.1. Background
8.2. Interviews/Summaries
Dr. Mick W. Graham, Principal Scientist, Benitec
Dr. Brian Johnston, President and CEO, SomaGenics Inc., and Consulting Associate
Professor of Pediatrics, Stanford University School of Medicine
Dr. Sujay Singh, President and CEO, Imgenex Corporation
John Rossi, City of Hope
Dr. Barry Polisky, Senior Vice President of Research and CSO, Sirna Therapeutics
Dr. Jean-Paul Reynes, Chief Scientist, siRNA Project, InvivoGen
Dr. Henrik Orum, Vice President and CSO, Santaris Pharma
Dr. Sam Reich, Vice President of Research and Development, Acuity Pharmaceuticals
Chapter 9. Concluding Remarks
Acknowledgments
References
Suggested Reading
Glossary
Index
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