Contributors
Preface
1 Milestones and Events in the Early Development of Human Gene Therapy
I. Phase 1. Prior to 1970: Genetically Marked Cells and DNA-Mediated Transformation
II. Phase 2. The Cline Experiment, Efficient Vectors, In Vitro Models, and the Maturing Ethical and Public Policy Debate
III. Phase 3. The Regulatory Process and the First Direct Clinical Applications: The Recombinant Advisory Committee (RAC), Its Gene Therapy Subcommittee, and the Food and Drug Administration (FDA)
References
2 Molecular Biology of Cystic Fibrosis
I. Introduction and History
II. Identification of the CF Gene
III. Mechanism of CFTR Function
IV. Genotype/Phenotype Relationships
V. Carrier Screening
VI. Therapeutic Prospects
VII. Summary
References
3 Genetics of Astrocytic Tumor Progression
I. Cancer is a Genetic Disease
II. Multistep Carcinogenesis
III. Tumor Progression
IV. Phenotypes of the Malignant Process
V. Mechanisms in Tumor Progression
VI. Clinical-Pathologic Correlation
VII. Biology of Astrocytic Tumors
VIII. Genetics of Astrocytic Tumors
IX. A Clonal Evolution Model for Astrocytic Tumors
X. Testing the Genetic Model
XI. Conclusion
References
4 Molecular Biology of Alzheimer's Disease
I. Introduction
II. History
III. Epidemiology
IV. Genetics
V. Clinical and Neuropsychological Features
VI. Diagnosis
VII. Treatment
VIII. Nerve Growth Factor and Related Molecules in the Central Nervous System
IX. Central Actions of Nerve Growth Factor
X. NGF-Related Trophic Factors: The Neurotrophins
XI. Neurotrophin Receptors
XII. NGF Mechanism of Action
XIII. A Model of Trophic Factor Action in Neurodegenerative Disease
XIV. Molecular Neuropathology
XV. Amyloid Deposition in Alzheimer's Disease
XVI. βΑΡΡ Processing
XVII. Conclusion: Clinical and Societal Implications
References
5 Hunting for Huntington's Disease
I. Introduction
II. Finding a Landmark near HD
III. Which Way to the Prey?
IV. Physical Layout of the Search Area
V. Tools for Tracking
VI. Limiting the Hunt Using Crossovers in Disease Pedigrees
VII. The Historical Trail of HD in Allele Associations
VIII. What is the Nature of the Quarry?
References
Note Added in Proof: Hunting's Done!
6 Genetics, Insurance, and the Ethics of Genetic Counseling
I. Introduction: Genetic Prediction and the Challenge to Insurance
II. Health Insurance
III. Life Insurance
IV. Ethical Conflicts of Providers of Genetic Services
V. Conclusion
References
Index