Delivery technologies for in utero gene therapy.
Delivery technologies for in utero gene therapy.
Adv Drug Deliv Rev. 2020 Nov 09;:
Authors: Palanki R, Peranteau WH, Mitchell MJ
Abstract
Advances in prenatal imaging, molecular diagnostic tools, and genetic screening have unlocked the possibility to treat congenital diseases in utero prior to the onset of clinical symptoms. While fetal surgery and in utero stem cell transplantation can be harnessed to treat specific structural birth defects and congenital hematological disorders, respectively, in utero gene therapy allows for phenotype correction of a wide range of genetic disorders within the womb. However, key challenges to realizing the broad potential of in utero gene therapy are biocompatibility and efficiency of intracellular delivery of transgenes. In this review, we outline the unique considerations to delivery of in utero gene therapy components and highlight advances in viral and non-viral delivery platforms that meet these challenges. We also discuss specialized delivery technologies for in utero gene editing and provide future directions to engineer novel delivery modalities for clinical translation of this promising therapeutic approach.
PMID: 33181188 [PubMed - as supplied by publisher]
Source: Advanced Drug Delivery Reviews - Category: Drugs & Pharmacology Authors: Palanki R, Peranteau WH, Mitchell MJ Tags: Adv Drug Deliv Rev Source Type: research
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