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Cited by 64Virus-based gene therapy researchRNA Interference and Gene DeliveryCRISPR and Genetic Engineering

Delivery technologies for in utero gene therapy

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Palanki, Rohan, Peranteau, William H., Mitchell, Michael J.·2021·Advanced Drug Delivery Reviews
DOI 10.1016/j.addr.2020.11.002PMC PMC7855052
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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.

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  • In utero gene editing takes advantage of normal fetal ontogeny to deliver therapies in a potentially more efficient manner, mitigating genetic disease before pathologic insult.

    “In utero gene editing takes advantage of normal fetal ontogeny to deliver therapies in a potentially more efficient manner, mitigating genetic disease before pathologic insult.”

    From Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease (2023) · cites this paper as [4]

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Authors
Palanki, R., Peranteau, W. H., & Mitchell, M. J.
Affiliations
  • University of Pennsylvania (US)
  • Children's Hospital of Philadelphia (US)
  • Abramson Cancer Center (US)
Corresponding author
Mitchell, Michael J.
ORCID
Venue
Advanced Drug Delivery Reviews
Publisher
Elsevier BV
Published
2021-02
Volume
169
Pages
51-62
ISSN
0169-409X (ISSN-L)
DOI
10.1016/j.addr.2020.11.002
PMC
PMC7855052
OpenAlex
W3098908552
Cited by
64
References
20
Citations by year
2026: 7 · 2025: 17 · 2024: 17 · 2023: 10 · 2022: 7 · 2021: 6
FWCI
0.83
Type
journal article
Language
en
Open access
Licence
Locations
  • Advanced Drug Delivery Reviews (publishedVersion)
  • PubMed (publishedVersion)
  • PubMed Central (acceptedVersion)
Funders
  • National Cancer Institute (R37 CA244911, R01 CA241661)
  • Harvard School of Engineering and Applied Sciences (P30 CA016520)
  • National Heart, Lung, and Blood Institute (DP2HL152427, R01HL151352)
  • National Center for Advancing Translational Sciences (DP2 TR002776)
  • Burroughs Wellcome Fund
  • Canadian Aeronautics and Space Institute
  • National Institute of Diabetes and Digestive and Kidney Diseases (R01DK123049)
  • Abramson Cancer Center
  • National Institutes of Health
  • American Cancer Society (129784- IRG-16-188-38-IRG)
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
Keywords
in utero gene therapy, gene delivery, viral vectors, non-viral vectors, genome editing, fetal development, biocompatibility, prenatal diagnosis, clinical translation
MeSH terms
Gene Editing, Animals, Fetal Diseases, Humans, Viruses, Genetic Therapy, Gene Transfer Techniques, Fetal Therapies

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

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