دورية أكاديمية
أعرض في EDS

Gene modification therapies for hereditary diseases in the fetus.

التفاصيل البيبلوغرافية
العنوان: Gene modification therapies for hereditary diseases in the fetus.
المؤلفون: Mattar CNZ; Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Singapore.; National University Health Systems, Singapore, Singapore., Chan JKY; KK Women's and Children's Hospital, Singapore, Singapore.; Duke-NUS Medical School, Singapore, Singapore., Choolani M; Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Singapore.; National University Health Systems, Singapore, Singapore.
المصدر: Prenatal diagnosis [Prenat Diagn] 2023 May; Vol. 43 (5), pp. 674-686. Date of Electronic Publication: 2023 Apr 05.
نوع المنشور: Journal Article; Review
اللغة: English
بيانات الدورية: Publisher: Wiley Country of Publication: England NLM ID: 8106540 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-0223 (Electronic) Linking ISSN: 01973851 NLM ISO Abbreviation: Prenat Diagn Subsets: MEDLINE
أسماء مطبوعة: Original Publication: Chichester, [Sussex]; New York : Wiley, c1981-
مواضيع طبية MeSH: Gene Transfer Techniques* , Gene Editing*, Humans ; Genetic Vectors ; Genetic Therapy ; Fetus
مستخلص: Proof-of-principle disease models have demonstrated the feasibility of an intrauterine gene modification therapy (in utero gene therapy (IUGT)) approach to hereditary diseases as diverse as coagulation disorders, haemoglobinopathies, neurogenetic disorders, congenital metabolic, and pulmonary diseases. Gene addition, which requires the delivery of an integrating or episomal transgene to the target cell nucleus to be transcribed, and gene editing, where the mutation is corrected within the gene of origin, have both been used successfully to increase normal protein production in a bid to reverse or arrest pathology in utero. While most experimental models have employed lentiviral, adenoviral, and adeno-associated viral vectors engineered to efficiently enter target cells, newer models have also demonstrated the applicability of non-viral lipid nanoparticles. Amelioration of pathology is dependent primarily on achieving sustained therapeutic transgene expression, silencing of transgene expression, production of neutralising antibodies, the dilutional effect of the recipient's growth on the mass of transduced cells, and the degree of pre-existing cellular damage. Safety assessment of any IUGT strategy will require long-term postnatal surveillance of both the fetal recipient and the maternal bystander for cell and genome toxicity, oncogenic potential, immune-responsiveness, and germline mutation. In this review, we discuss advances in the field and the push toward clinical translation of IUGT.
(© 2023 The Authors. Prenatal Diagnosis published by John Wiley & Sons Ltd.)
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معلومات مُعتمدة: MOH000672 National Medical Research Council; MOH00066801 National Medical Research Council; NMRC/CSASI/0008/2016 National Medical Research Council
تواريخ الأحداث: Date Created: 20230325 Date Completed: 20230511 Latest Revision: 20240320
رمز التحديث: 20240320
مُعرف محوري في PubMed: PMC10946994
DOI: 10.1002/pd.6347
PMID: 36965009
قاعدة البيانات: MEDLINE
الوصف
تدمد:1097-0223
DOI:10.1002/pd.6347