Human Germline Genome Editing

Ormond, Kelly E.; Mortlock, Douglas P.; Scholes, Derek T.; Bombard, Yvonne; Brody, Lawrence C.; Faucett, W. Andrew; Garrison, Nanibaa’ A.; Hercher, Laura; Isasi, Rosario; Middleton, Anna; Musunuru, Kiran; Shriner, Daniel; Virani, Alice; Young, Caroline E.

doi:10.1016/j.ajhg.2017.06.012

Cited by 184 publications

(134 citation statements)

References 51 publications

(41 reference statements)

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“…This is notable because numerous official statements have also endorsed such research, including ones from the U.S. National Academies of Sciences, Engineering, and Medicine 6 and the American Society of Human Genetics. 7 A substantial majority of our respondents also supported public (government) funding of in vitro research that would not culminate in pregnancy. Although this is probably a non-starter in the U.S. for the foreseeable future, it accords with the call from the American Society of Human Genetics that there be no prohibition on public funding of in vitro research.…”

mentioning

confidence: 90%

“…Although this is probably a non-starter in the U.S. for the foreseeable future, it accords with the call from the American Society of Human Genetics that there be no prohibition on public funding of in vitro research. 7 …”

mentioning

confidence: 99%

“…Nonetheless, we felt that the plenary session was an example of a meaningful consultation with an important constituency, one that could be replicated in some format in many settings with many different constituencies. In light of the American Society of Human Genetics statement’s call for “a transparent public process to solicit and incorporate stakeholder input,” 7 we urge health advocacy organizations like the American Heart Association to foster and participate in that process.…”

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confidence: 99%

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What Do We Really Think About Human Germline Genome Editing, and What Does It Mean for Medicine?

Musunuru

Lagor

Miano

2017

Circ Cardiovasc Genet

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Genome editing has captured widespread attention due to its potential therapeutic applications. Early studies with human embryos have established the feasibility of human germline genome editing but raise complex social, ethical, and legal questions. In light of the potential impact of genome editing on the practice of cardiovascular medicine, we surveyed ≈300 attendees at a recent American Heart Association conference to elicit their opinions on somatic and germline genome editing. The results were revealing and highlight the need to broadly engage the public and solicit the opinions of various constituencies before proceeding with clinical germline genome editing.

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confidence: 90%

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confidence: 99%

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confidence: 99%

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What Do We Really Think About Human Germline Genome Editing, and What Does It Mean for Medicine?

Musunuru

Lagor

Miano

2017

Circ Cardiovasc Genet

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“…Germline gene editing is defined as the modification of gametes (i.e. oocyte and sperm), zygote and embryo for either therapeutic or non‐therapeutic reasons using gene editing technology such as CRISPR‐Cas9 and others . Germline gene editing results in the inheritable change of targeted genetic traits of the offspring of the parental donors.…”

Section: Germline Gene Editingmentioning

confidence: 99%

Gene editing of stem cells for kidney disease modelling and therapeutic intervention

2018

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Recent developments in targeted gene editing have paved the way for the wide adoption of clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein-9 nucleases (Cas9) as an RNA-guided molecular tool to modify the genome of eukaryotic cells of animals. Theoretically, the translation of CRISPR-Cas9 can be applied to the treatment of inherited or acquired kidney disease, kidney transplantation and genetic corrections of somatic cells from kidneys with inherited mutations, such as polycystic kidney disease. Human pluripotent stem cells have been used to generate an unlimited source of kidney progenitor cells or, when spontaneously differentiated into three-dimensional kidney organoids, to model kidney organogenesis or the pathogenesis of disease. Gene editing now allows for the tagging and selection of specific kidney cell types or disease-specific gene knock in/out, which enables more precise understanding of kidney organogenesis and genetic diseases. This review discusses the mechanisms of action, in addition to the advantages and disadvantages, of the three major gene editing technologies, namely, CRISPR-Cas9, zinc finger nucleases and transcription activator-like effector nucleases. The implications of using gene editing to better understand kidney disease is reviewed in detail. In addition, the ethical issues of gene editing, which could be easily neglected in the modern, fast-paced research environment, are highlighted.

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“…The U.S. National Academy of Sciences and National Academy of Medicine hosted an International Summit on Human Gene Editing, and its final published report supported basic science research using genome editing of human embryos to study reproductive biology, as long as it is performed within an appropriate regulatory framework (111). The American Society of Human Genetics statement on human germline genome editing went a step further in saying that there is “no reason to prohibit in vitro germline genome editing on human embryos and gametes, with appropriate oversight and consent from donors, to facilitate research on the possible future clinical applications of gene editing” and there should be “no prohibition on making public funds available to support this research” (112). …”

Section: Human Germline Genome Editingmentioning

confidence: 99%

Genome Editing

Musunuru

2017

Journal of the American College of Cardiology

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The genome editing field has advanced to a remarkable degree in the last five years, culminating in the successful correction of a cardiomyopathy gene mutation in viable human embryos. In this review, I discuss the basic principles of genome editing, recent advances in clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated 9 (Cas9) technology, the impact on cardiovascular basic science research, possible therapeutic applications in patients with cardiovascular diseases, and finally the implications of potential clinical uses of human germline genome editing.

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Human Germline Genome Editing

Cited by 184 publications

References 51 publications

What Do We Really Think About Human Germline Genome Editing, and What Does It Mean for Medicine?

What Do We Really Think About Human Germline Genome Editing, and What Does It Mean for Medicine?

Gene editing of stem cells for kidney disease modelling and therapeutic intervention

Genome Editing

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