Prospects for prenatal gene therapy in disorders causing mental retardation

Ye, Xuehai; Mitchell, Melanie; Newman, Kurt D.; Batshaw, Mark L.

doi:10.1002/1098-2779(200102)7:1<65::aid-mrdd1009>3.0.co;2-9

Cited by 14 publications

(11 citation statements)

References 59 publications

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“…Many frameworks propose categorical requirements for all studies irrespective of their distinctive features. Several frameworks require, for example, that anticipated fetal benefits be above a certain threshold (e.g., only interventions with the prospect of alleviating life‐threatening or serious and irreversible diseases) 3, 9, 11, 12, 14–25 . Absolute risk limits for pregnant persons are also common (e.g., low or manageable risks) 11, 12, 15–19, 21, 26–28 .…”

Section: Existing Ethical Frameworkmentioning

confidence: 99%

A new ethical framework to determine acceptable risks in fetal therapy trials

et al. 2022

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Objective Fetal therapy trials pose complex ethical challenges because risks and benefits to both fetuses and pregnant persons must be considered. Existing regulatory guidance is limited and many proposed ethical frameworks have unnecessarily restrictive criteria that would block the development and implementation of important new fetal therapies. We aimed to develop a new ethical framework for assessing the risks and benefits of fetal therapy trials. Methods We reviewed existing regulatory and ethical guidance on fetal therapy trials. We used conceptual analysis to design a new ethical framework, which is grounded in general ethical principles for clinical research. Results We propose a new framework for assessing the risks and benefits of fetal therapy trials. We suggest that the potential benefits of a fetal therapy trial – for the fetus, the pregnant person, and society – should outweigh the risks for the fetus and the pregnant person. Furthermore, the risk‐benefit profile for just the fetus and the risk‐benefit profile for just the pregnant person should be appropriate. Conclusions We hope that this new framework will permit important studies while protecting pregnant persons and fetuses from disproportionate harms.

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Section: Existing Ethical Frameworkmentioning

confidence: 99%

A new ethical framework to determine acceptable risks in fetal therapy trials

et al. 2022

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“…Much of the debate involved prenatal diagnosis of severe malformations (Bayley, 1980;Friedmann, 1971). One writer has held out the promise of gene therapy to correct several problems of prenatal life thus avoiding such difficult ethical questions about possible euthanasia (Ye, Mitchell, Newman, & Batshaw, 2001).…”

Section: Euthanasiamentioning

confidence: 99%

Handbook of Intellectual and Developmental Disabilities

Jacobson¹,

Mulick²,

Rojahn³

2007

Issues on Clinical Child Psychology

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“…Gene therapy raises the possibility of introducing genetic modifications into the recipient’s germ cells, which could then be passed on to future generations. Studies suggest a low risk of germ line transmission [82]. …”

Section: Introductionmentioning

confidence: 99%

Integration of maternal genome into the neonate genome through breast milk mRNA transcripts and reverse transcriptase

Irmak

Öztaş

2012

Theor Biol Med Model

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Human milk samples contain microvesicles similar to the retroviruses. These microvesicles contain mRNA transcripts and possess reverse transcriptase activity. They contain about 14,000 transcripts representing the milk transcriptome. Microvesicles are also enriched with proteins related to “caveolar-mediated endocytosis signaling” pathway. It has recently been reported that microvesicles could be transferred to other cells by endocytosis and their RNA content can be translated and be functional in their new location. A significant percentage of the mammalian genome appears to be the product of reverse transcription, containing sequences whose characteristics point to RNA as a template precursor. These are mobile elements that move by way of transposition and are called retrotransposons. We thought that retrotransposons may stem from about 14,000 transcriptome of breast milk microvesicles, and reviewed the literature. The enhanced acceptance of maternal allografts in children who were breast-fed and tolerance to the maternal MHC antigens after breastfeeding may stem from RNAs of the breast milk microvesicles that can be taken up by the breastfed infant and receiving maternal genomic information. We conclude that milk microvesicles may transfer genetic signals from mother to neonate during breastfeeding. Moreover, transfer of wild type RNA from a healthy wet-nurse to the suckling neonate through the milk microvesicles and its subsequent reverse transcription and integration into the neonate genome could result in permanent correction of the clinical manifestations in genetic diseases.

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Prospects for prenatal gene therapy in disorders causing mental retardation

Cited by 14 publications

References 59 publications

A new ethical framework to determine acceptable risks in fetal therapy trials

A new ethical framework to determine acceptable risks in fetal therapy trials

Handbook of Intellectual and Developmental Disabilities

Integration of maternal genome into the neonate genome through breast milk mRNA transcripts and reverse transcriptase

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