Protocol

Prenatal Gene Therapy

Volume 891 of the series Methods in Molecular Biology pp 169-181

Date:

Use of Manipulated Stem Cells for Prenatal Therapy

  • Jessica L. RoybalAffiliated withDepartment of Surgery, Children’s Center for Fetal Research and Center for Fetal Diagnosis and Treatment, Children’s Hospital of Philadelphia
  • , Pablo LajeAffiliated withDepartment of Surgery, Children’s Center for Fetal Research and Center for Fetal Diagnosis and Treatment, Children’s Hospital of Philadelphia
  • , Jesse D. VrecenakAffiliated withDepartment of Surgery, Children’s Center for Fetal Research and Center for Fetal Diagnosis and Treatment, Children’s Hospital of Philadelphia
  • , Alan W. FlakeAffiliated withDepartment of Surgery, Children’s Hospital of Philadelphia Email author 

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Abstract

Prenatal stem cell therapy has broad potential for therapeutic application. “Stem cells” of interest include multipotent adult-derived stem cells, cord blood, amniotic fluid, or fetal stem cells, and embryonic or induced pluripotent stem cells. Potential manipulations of stem cells prior to their administration may include harvest, processing, enrichment, expansion, and genetic transduction. A complete description of the methodology related to all of the above is well beyond the scope of this chapter. In the interest of practical application and proven efficacy, we limit our description to adult-derived hematopoietic stem cells (HSCs) and their application to in utero transplantation with or without HSC-targeted gene transfer.

Key words

In utero transplantation Hematopoietic stem cell Lentiviral vector Gene transfer