Organ targeted prenatal gene therapy—how far are we?

No abstract is available for this paper.

• Publication year

  2011

• Venue

  Prenatal Diagnosis

• Publication date

  2011-07-01

• Fields of study

  Medicine

• Identifiers
  DOI 10.1002/pd.2787PMID 21618255
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

• Prenatal Gene Therapy

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• Lysosomal Storage Disorders

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• Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells

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• Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state

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• Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.

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• Fetal Gene Transfer Using Lentiviral Vectors: In Vivo Detection of Gene Expression by microPET and Optical Imaging in Fetal and Infant Monkeys

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• Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors.

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• Clinically Applicable Procedure for Gene Delivery to Fetal Gut by Ultrasound-Guided Gastric Injection: Toward Prenatal Prevention of Early-Onset Intestinal Diseases

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• Improvement of pulmonary hypoplasia associated with congenital diaphragmatic hernia by in utero CFTR gene therapy.

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• Targeting the respiratory muscles of fetal sheep for prenatal gene therapy for Duchenne muscular dystrophy.

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• Placental mesenchymal stem cells as potential autologous graft for pre- and perinatal neuroregeneration.

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• Factors influencing adenovirus-mediated airway transduction in fetal mice.

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  2005influential reference
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  2004influential reference
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• Long-term correction of bilirubin UDPglucuronyltransferase deficiency in rats by in utero lentiviral gene transfer.

  2003cited by this paper
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  2003cited by this paper
• Transgenes encompassing dual-promoter CpG islands from the human TBP and HNRPA2B1 loci are resistant to heterochromatin-mediated silencing.

  2003influential reference
• A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer.

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• Enhancement of adenovirus-mediated gene transfer to the airways by DEAE dextran and sodium caprate in vivo.

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• Fetal drug therapy

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• Intrauterine Fetal Gene Therapy: Is That the Future and Is That Future Now?

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• Fetal gene therapy: Balancing ethical theory, scientific progress and the rights of others

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• Perinatal Gene Transfer to the Liver

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