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Comment
. 2018 Sep 6;23(3):320-321.
doi: 10.1016/j.stem.2018.08.010.

Future AAVenues for In Utero Gene Therapy

Tippi C MacKenzie  1
Affiliations
Free article
Comment

Future AAVenues for In Utero Gene Therapy

Tippi C MacKenzie. Cell Stem Cell. .
Free article

Abstract

Fetal gene therapy using safe and effective viral vectors no longer remains a distant prospect. Recently in Nature Medicine, Massaro et al. (2018) demonstrated that prenatal intracranial injection of a viral vector results in improved neurologic function, raising the intriguing possibility that in utero gene therapy may be approaching clinical applications.

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Comment on

  • Fetal gene therapy for neurodegenerative disease of infants.
    Massaro G, Mattar CNZ, Wong AMS, Sirka E, Buckley SMK, Herbert BR, Karlsson S, Perocheau DP, Burke D, Heales S, Richard-Londt A, Brandner S, Huebecker M, Priestman DA, Platt FM, Mills K, Biswas A, Cooper JD, Chan JKY, Cheng SH, Waddington SN, Rahim AA. Massaro G, et al. Nat Med. 2018 Sep;24(9):1317-1323. doi: 10.1038/s41591-018-0106-7. Epub 2018 Jul 16. Nat Med. 2018. PMID: 30013199 Free PMC article.

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