10-12 Not yet established

Maternal blood

Skin, bladder, digestive system cells in amniotic fluid Chorionic villi Maternal blood

Small liver may indicate increased risk of trisomy Fetal karyotype

Fetal karyotype Fetal karyotype

Requirements for Approval of Clinical Trials for Gene Therapy

1. Knowledge of defect and how it causes symptoms.

2. An animal model.

3. Success in human cells growing in vitro.

4. Either no alternate therapies, or a group of patients for whom existing therapies are not possible or have not worked.

5. Experiments must be safe.

┬╗Gene Therapy Concerns

1. Which cells should be treated?

2. What proportion of the targeted cell population must be corrected to alleviate or halt progression of symptoms?

3. Is overexpression of the therapeutic gene dangerous?

4. If the engineered gene "escapes" and infiltrates other tissues, is there danger?

5. How long will the affected cells function?

6. Will the immune system attack the introduced cells?

patients, whereas gene therapy for CF so far has not provided a lasting cure. The age of gene therapy began with success, but recently hit a setback, discussed in Clinical Application 24.3. Tables 24.2 and 24.3 list guidelines for gene therapy trials.

Essentials of Human Physiology

Essentials of Human Physiology

This ebook provides an introductory explanation of the workings of the human body, with an effort to draw connections between the body systems and explain their interdependencies. A framework for the book is homeostasis and how the body maintains balance within each system. This is intended as a first introduction to physiology for a college-level course.

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