SECTION I Disease Classification and Possible Diagnosis 1. Clinical Features to Consider in a Patient with Possible Genetic Disease 2. Determining the Presence Of Altered Values in Clinical Laboratory Studies 3. Further Laboratory-Based Investigations for Possible Gene or Genome Based Disorders SECTION 2A: Application of Therapies and Strategies for Development of Therapies 4. Review Possible Therapies to Address Clinical Manifestations Including Symptoms and Signs and Abnormal Results of Metabolic, Biochemical, Gene Based, Microarray or Chromosome Studies 5. Review Investigations of Possible Therapies to Address Underlying Disease-Related Functional Impairments, Possible Known Genetic Disorders, and Review Reports of Established Therapies for Specific Genetic Diseases SECTION 2B. Discussion of Specific Diseases Where Gene Therapy, Gene-Based Therapies or Small Molecule-Based Therapies Have Been Successful 6. Examples of Diseases Where Appropriate Small Molecule Therapies Were Discovered 7.
Therapies That Address Altered Gene Regulation SECTION 3A Review Research on Therapeutic Design 8. Review Research Designed to Investigate Gene Function and Possible Impact of Specific Variants 9. Designing Therapies Relevant in Human Genetic Disorders SECTION 3B 10. Clinical Trial Designs and Permissions.