Toxicology and Biodistribution: The Clinical Value of Animal Biodistribution Studies
Since the human genome decoding, understanding and identification of genetic disturbances behind many diseases, including cancer, are intensively increasing. Scientific and technological advances in this area trigger the search for therapeutic (curative) approaches targeting the correction of gene d...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
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Elsevier
2018-03-01
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| Series: | Molecular Therapy: Methods & Clinical Development |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050118300044 |
| _version_ | 1811238720901218304 |
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| author | Beatriz Silva Lima Mafalda Ascensão Videira |
| author_facet | Beatriz Silva Lima Mafalda Ascensão Videira |
| author_sort | Beatriz Silva Lima |
| collection | DOAJ |
| description | Since the human genome decoding, understanding and identification of genetic disturbances behind many diseases, including cancer, are intensively increasing. Scientific and technological advances in this area trigger the search for therapeutic (curative) approaches targeting the correction of gene disturbances. Gene therapy medicinal products (GTMPs) emerge in this context, bringing new challenges for their characterization. Compared to small molecules, biodistribution is fundamental to identifying target organs and anticipating safety and efficacy, may be integrated into safety and pharmacology studies, and may eventually be anticipated based on specificities of vectors and constructs. This review describes and discusses the requirements for nonclinical development and evaluation of GTMPs versus conventional ones and the needs and challenges of constructing nonclinical packages that assure GTMPs’ human safety from early development, taking into consideration usefulness and/or limitations of many conventional, preclinical models. The experience gained in the European context is referenced. |
| first_indexed | 2024-04-12T12:45:58Z |
| format | Article |
| id | doaj.art-78015c6b1680424f8312e14d6dc6af6f |
| institution | Directory Open Access Journal |
| issn | 2329-0501 |
| language | English |
| last_indexed | 2024-04-12T12:45:58Z |
| publishDate | 2018-03-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Molecular Therapy: Methods & Clinical Development |
| spelling | doaj.art-78015c6b1680424f8312e14d6dc6af6f2022-12-22T03:32:36ZengElsevierMolecular Therapy: Methods & Clinical Development2329-05012018-03-018C18319710.1016/j.omtm.2018.01.003Toxicology and Biodistribution: The Clinical Value of Animal Biodistribution StudiesBeatriz Silva Lima0Mafalda Ascensão Videira1Universidade de Lisboa, Faculdade de Farmácia, iMED.ULisboa, Lisboa 1649003, PortugalUniversidade de Lisboa, Faculdade de Farmácia, iMED.ULisboa, Lisboa 1649003, PortugalSince the human genome decoding, understanding and identification of genetic disturbances behind many diseases, including cancer, are intensively increasing. Scientific and technological advances in this area trigger the search for therapeutic (curative) approaches targeting the correction of gene disturbances. Gene therapy medicinal products (GTMPs) emerge in this context, bringing new challenges for their characterization. Compared to small molecules, biodistribution is fundamental to identifying target organs and anticipating safety and efficacy, may be integrated into safety and pharmacology studies, and may eventually be anticipated based on specificities of vectors and constructs. This review describes and discusses the requirements for nonclinical development and evaluation of GTMPs versus conventional ones and the needs and challenges of constructing nonclinical packages that assure GTMPs’ human safety from early development, taking into consideration usefulness and/or limitations of many conventional, preclinical models. The experience gained in the European context is referenced.http://www.sciencedirect.com/science/article/pii/S2329050118300044gene therapyrisk-based approachpreclinical programsbiodistributionmodel relevanceadvanced therapy medicinal productsATMPs |
| spellingShingle | Beatriz Silva Lima Mafalda Ascensão Videira Toxicology and Biodistribution: The Clinical Value of Animal Biodistribution Studies Molecular Therapy: Methods & Clinical Development gene therapy risk-based approach preclinical programs biodistribution model relevance advanced therapy medicinal products ATMPs |
| title | Toxicology and Biodistribution: The Clinical Value of Animal Biodistribution Studies |
| title_full | Toxicology and Biodistribution: The Clinical Value of Animal Biodistribution Studies |
| title_fullStr | Toxicology and Biodistribution: The Clinical Value of Animal Biodistribution Studies |
| title_full_unstemmed | Toxicology and Biodistribution: The Clinical Value of Animal Biodistribution Studies |
| title_short | Toxicology and Biodistribution: The Clinical Value of Animal Biodistribution Studies |
| title_sort | toxicology and biodistribution the clinical value of animal biodistribution studies |
| topic | gene therapy risk-based approach preclinical programs biodistribution model relevance advanced therapy medicinal products ATMPs |
| url | http://www.sciencedirect.com/science/article/pii/S2329050118300044 |
| work_keys_str_mv | AT beatrizsilvalima toxicologyandbiodistributiontheclinicalvalueofanimalbiodistributionstudies AT mafaldaascensaovideira toxicologyandbiodistributiontheclinicalvalueofanimalbiodistributionstudies |