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Prenatal Gene Therapy for Metabolic Disorders

Barbara Coons¹, William H Peranteau

¹Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.

Clinical Obstetrics and Gynecology

|October 15, 2021

Summary

Gene therapy delivers genetic material to correct disorders. Advanced gene editing tools like CRISPR, TALENs, and ZFNs now offer precise genetic modification for therapeutic purposes.

Area of Science:

Biotechnology
Molecular Biology
Genetics

Background:

Gene therapy traditionally replaces defective genes to restore protein production for treating monogenic disorders.
The field has evolved significantly over 40 years.

Purpose of the Study:

To provide an overview of the evolution of gene therapy.
To highlight the expansion into targeted gene editing strategies.

Main Methods:

Review of traditional gene therapy delivery methods.
Introduction to gene editing technologies such as CRISPR/Cas9, TALENs, and ZFNs.

Main Results:

Gene therapy has expanded beyond simple gene replacement.
Targeted gene editing offers new therapeutic possibilities.

Conclusions:

Gene therapy has advanced to include sophisticated gene editing tools.
These technologies represent a significant expansion in therapeutic potential.

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