Adenovirus-mediated prenatal gene transfer to murine central nervous system

Js, Shen; Xl, Meng; Ohashi, Toya; Eto, Yoshikatsu

doi:10.1038/sj.gt.3301700

Cited by 11 publications

(2 citation statements)

References 23 publications

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“…In utero gene delivery has been investigated in animal models and target genes have been transduced into visceral organs and hematopoietic cells 8–12. There have been other studies including ours relating to gene delivery in utero to the CNS through systemic circulation 13 or amniotic cavity 14. However, there was limited gene transfer to the neural cells and no evidence showed the therapeutic ability of these approaches in global brain diseases.…”

Section: Introductionmentioning

confidence: 99%

Widespread gene transduction to the central nervous system by adenovirus in utero: implication for prenatal gene therapy to brain involvement of lysosomal storage disease

Shen

Meng

Maéda

et al. 2004

The Journal of Gene Medicine

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Section: Introductionmentioning

confidence: 99%

Widespread gene transduction to the central nervous system by adenovirus in utero: implication for prenatal gene therapy to brain involvement of lysosomal storage disease

Shen

Meng

Maéda

et al. 2004

The Journal of Gene Medicine

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“…Shen and colleagues performed a series of studies investigating adenovirus vectors for gene transfer to the rat CNS, first by intra-amniotic injection at mid-gestation (12 dpc, where dpc is days postcoitus; gestation in mice and rats is approx. 20 days) [13] and then, more targeted, by injection into the lateral ventricle at late gestation (14 and 17 dpc) [14]. The late gestation injection, in particular, led to long-term expression for up to 10 months after injection, albeit at diminishing levels.…”

Section: Gene Transfer As a Potential Therapymentioning

confidence: 97%

In utero gene transfer to the mouse nervous system

Rahim

Wong

Buckley

et al. 2010

Biochemical Society Transactions

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The cellular and molecular environment present in the fetus and early newborn provides an excellent opportunity for effective gene transfer. Innate and pre-existing anti-vector immunity may be attenuated or absent and the adaptive immune system predisposed to tolerance towards xenoproteins. Stem cell and progenitor cell populations are abundant, active and accessible. In addition, for treatment of early lethal genetic diseases of the nervous system, the overarching advantage may be that early gene supplementation prevents the onset of irreversible pathological changes. Gene transfer to the fetal mouse nervous system was achieved, albeit inefficiently, as far back as the mid-1980s. Recently, improvements in vector design and production have culminated in near-complete correction of a mouse model of spinal muscular atrophy. In the present article, we review perinatal gene transfer from both a therapeutic and technological perspective.

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Organ targeted prenatal gene therapy—how far are we?

Mehta¹,

Nader²,

Waddington³

et al. 2011

Prenatal Diagnosis

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Prenatal gene therapy aims to deliver genes to cells and tissues early in prenatal life, allowing correction of a genetic defect, before long-term tissue damage has occurred. In contrast to postnatal gene therapy, prenatal application can target genes to a large population of dividing stem cells, and the smaller fetal size allows a higher vector-to-target cell ratio to be achieved. Early-gestation delivery may allow the development of immune tolerance to the transgenic protein which would facilitate postnatal repeat vector administration if needed. Targeting particular organs will depend on manipulating the vector to achieve selective tropism and on choosing the most appropriate gestational age and injection method for fetal delivery. Intra-amniotic injection reaches the skin, and other organs that are bathed in the fluid however since gene transfer to the lung and gut is usually poor more direct injection methods will be needed. Delivery to the liver and blood can be achieved by systemic delivery via the umbilical vein or peritoneal cavity. Gene transfer to the central nervous system in the fetus is difficult but newer vectors are available that transduce neuronal tissue even after systemic delivery.

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Adenovirus-mediated prenatal gene transfer to murine central nervous system

Cited by 11 publications

References 23 publications

Widespread gene transduction to the central nervous system by adenovirus in utero: implication for prenatal gene therapy to brain involvement of lysosomal storage disease

Widespread gene transduction to the central nervous system by adenovirus in utero: implication for prenatal gene therapy to brain involvement of lysosomal storage disease

In utero gene transfer to the mouse nervous system

Organ targeted prenatal gene therapy—how far are we?

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