Evidence map›Paper›PMID 23103867›Full record

ArticleNature2013

Towards germline gene therapy of inherited mitochondrial diseases.

Masahito Tachibana, Paula Amato, Michelle Sparman, Joy Woodward, Dario Melguizo Sanchis, Hong Ma, Nuria Marti Gutierrez, Rebecca Tippner-Hedges, Eunju Kang, Hyo-Sang Lee and 10 more

Open access · greenAbstract read
In one paragraph

Article in Nature, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 166 papers.

0numbers the graph read from it
0cells of the map it votes in
166citing papers in PubMed
38.0field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

166 citing papers in PubMed, 421 citations in OpenAlex.

  1. Review
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  16. Review
  17. Nanoparticle-based Gene Therapy for Neurodegenerative Disorders.Mini reviews in medicinal chemistry · 2024
    Review
  18. Article
  19. Article
  20. Article

106 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

20 authors at 3 institutions in 1 country.

Masahito TachibanaDivision of Reproductive & Developmental Sciences, Oregon National Primate Research Center, Oregon Health & Science University, 505 NW 185th Avenue, Beaverton, Oregon 97006, USA.
Paula Amato
Michelle Sparman
Joy Woodward
Dario Melguizo Sanchis
Hong Ma
Nuria Marti Gutierrez
Rebecca Tippner-Hedges
Eunju Kang
Hyo-Sang Lee
Cathy Ramsey
Keith Masterson
David Battaglia
David Lee
Diana Wu
Jeffrey Jensen
Phillip Patton
Sumita Gokhale
Richard Stouffer
Shoukhrat Mitalipov
Oregon Health & Science University · USOregon National Primate Research Center · USRoger Williams Medical Center · US

Funding

Upgrade of confocal microscopy at the Oregon National Primate Research CenterP51OD011092 · OD · OREGON HEALTH & SCIENCE UNIVERSITY · PI Bonnie J. Nagel · 2012 to 2026
$203.9M
YR 50 PROJ- ROBERTS PRIMATE ISLETP51RR000163 · NCRR · OREGON REGIONAL PRIMATE RESEARCH CENTER · PI FRUH, KLAUS J · 1985 to 2011
$140.8M
Histocompatible Primate Embryonic Stem CellsR01HD057121 · NICHD · OREGON HEALTH & SCIENCE UNIVERSITY · PI MITALIPOV, SHOUKHRAT · 2009 to 2013
$3.4M
Mitochondrial Gene TherapyR01HD063276 · NICHD · OREGON HEALTH & SCIENCE UNIVERSITY · PI MITALIPOV, SHOUKHRAT · 2010 to 2014
$3.0M
Altered Nuclear TransferR01HD059946 · NICHD · OREGON HEALTH & SCIENCE UNIVERSITY · PI MITALIPOV, SHOUKHRAT · 2010 to 2014
$2.7M
Evaluation of stem cell-derived retinal pigment epithelial cells for retinal diseR01EY021214 · NEI · OREGON HEALTH & SCIENCE UNIVERSITY · PI NEURINGER, MARTHA · 2011 to 2015
$2.1M
NCRR NIH HHS P51 RR000163NEI NIH HHS EY021214NEI NIH HHS R01 EY021214NICHD NIH HHS HD057121NICHD NIH HHS HD059946NICHD NIH HHS HD063276NICHD NIH HHS R01 HD057121NICHD NIH HHS R01 HD059946NICHD NIH HHS R01 HD063276NIH HHS 8P51OD011092NIH HHS P51 OD011092
6 · The paper itself

Abstract

Mutations in mitochondrial DNA (mtDNA) are associated with severe human diseases and are maternally inherited through the egg's cytoplasm. Here we investigated the feasibility of mtDNA replacement in human oocytes by spindle transfer (ST; also called spindle-chromosomal complex transfer). Of 106 human oocytes donated for research, 65 were subjected to reciprocal ST and 33 served as controls. Fertilization rate in ST oocytes (73%) was similar to controls (75%); however, a significant portion of ST zygotes (52%) showed abnormal fertilization as determined by an irregular number of pronuclei. Among normally fertilized ST zygotes, blastocyst development (62%) and embryonic stem cell isolation (38%) rates were comparable to controls. All embryonic stem cell lines derived from ST zygotes had normal euploid karyotypes and contained exclusively donor mtDNA. The mtDNA can be efficiently replaced in human oocytes. Although some ST oocytes displayed abnormal fertilization, remaining embryos were capable of developing to blastocysts and producing embryonic stem cells similar to controls.

Indexed as

Genetic TherapyAdultAnimalsCell NucleusCryopreservationCytoplasmDNA, MitochondrialEmbryo, MammalianEmbryonic Stem CellsFemaleFertilizationHumansMacaca mulattaMicrosatellite RepeatsMitochondrial DiseasesNuclear Transfer TechniquesDNA, Mitochondrial

Identifiers

PMID23103867
PMCPMC3561483
OpenAlexW1963825171

What OpenQuestion holds

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LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.