Molecular Basis and Gene Therapies of Cystic Fibrosis (Record no. 46061)
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| fixed length control field | 04326naaaa2200961uu 4500 |
| 001 - CONTROL NUMBER | |
| control field | https://directory.doabooks.org/handle/20.500.12854/69404 |
| 005 - DATE AND TIME OF LATEST TRANSACTION | |
| control field | 20220219215046.0 |
| 020 ## - INTERNATIONAL STANDARD BOOK NUMBER | |
| International Standard Book Number | books978-3-03943-684-2 |
| 020 ## - INTERNATIONAL STANDARD BOOK NUMBER | |
| International Standard Book Number | 9783039436835 |
| 020 ## - INTERNATIONAL STANDARD BOOK NUMBER | |
| International Standard Book Number | 9783039436842 |
| 024 7# - OTHER STANDARD IDENTIFIER | |
| Standard number or code | 10.3390/books978-3-03943-684-2 |
| Terms of availability | doi |
| 041 0# - LANGUAGE CODE | |
| Language code of text/sound track or separate title | English |
| 042 ## - AUTHENTICATION CODE | |
| Authentication code | dc |
| 072 #7 - SUBJECT CATEGORY CODE | |
| Subject category code | M |
| Source | bicssc |
| 100 1# - MAIN ENTRY--PERSONAL NAME | |
| Personal name | Engelhardt, John |
| Relationship | edt |
| 245 10 - TITLE STATEMENT | |
| Title | Molecular Basis and Gene Therapies of Cystic Fibrosis |
| 260 ## - PUBLICATION, DISTRIBUTION, ETC. | |
| Place of publication, distribution, etc. | Basel, Switzerland |
| Name of publisher, distributor, etc. | MDPI - Multidisciplinary Digital Publishing Institute |
| Date of publication, distribution, etc. | 2020 |
| 300 ## - PHYSICAL DESCRIPTION | |
| Extent | 1 electronic resource (210 p.) |
| 506 0# - RESTRICTIONS ON ACCESS NOTE | |
| Terms governing access | Open Access |
| Source of term | star |
| Standardized terminology for access restriction | Unrestricted online access |
| 520 ## - SUMMARY, ETC. | |
| Summary, etc. | Summary of Genes. Thirty years ago, the gene responsible for cystic fibrosis (CF), a recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator gene, was identified. This progress has considerably changed our understanding of the pathophysiology of CF and has paved the way for the development of novel and specific therapies for the disease. The CFTR gene contains 27 exons and is characterized by a frequent three base pair deletion of the p.Phe508del. As a result of collaborative work, today more than 2000 mutations have been reported in the gene, and their impact on protein function is now more evident and useful in designing new strategies to correct the gene defect. The field of gene therapy, as illustrated by Ziying Yan in this book, has worked on identifying an efficient vector system for the delivery of the wild-type CFTR gene to the lung. At the same time, animal models have been developed in mice, rats, rabbits, zebrafish, ferrets, and pigs to establish the efficacity of gene delivery. These animals are also of the utmost importance in testing new molecules as modulators or correctors to improve the CFTR lung function. During the last three decades, the epidemiology of CF has dramatically changed, as today cystic fibrosis is now a chronic adult pulmonary disease. |
| 540 ## - TERMS GOVERNING USE AND REPRODUCTION NOTE | |
| Terms governing use and reproduction | Creative Commons |
| Use and reproduction rights | https://creativecommons.org/licenses/by/4.0/ |
| Source of term | cc |
| -- | https://creativecommons.org/licenses/by/4.0/ |
| 546 ## - LANGUAGE NOTE | |
| Language note | English |
| 650 #7 - SUBJECT ADDED ENTRY--TOPICAL TERM | |
| Topical term or geographic name entry element | Medicine |
| Source of heading or term | bicssc |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | cystic fibrosis |
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| Uncontrolled term | Staphylococcus aureus |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | superantigen |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | enterotoxin gene cluster |
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| Uncontrolled term | MRSA |
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| Uncontrolled term | exosomes |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | microvesicles |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | lung |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | primary cells |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | newborn screening |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | trypsinogen |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | CFTR gene |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | next generation sequencing |
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| Uncontrolled term | health policy |
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| Uncontrolled term | rAAV2/HBoV1 |
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| Uncontrolled term | baculovirus |
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| Uncontrolled term | insect cells |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | lung microbiome |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | metagenomics |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | gut–lung axis |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | Cystic fibrosis |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | CFTR |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | transcriptomics |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | proteostasis |
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| Uncontrolled term | small molecules |
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| Uncontrolled term | drug development |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | common and new pathogenic variants |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | ethnic Russian population |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | gene therapy |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | cyclophosphamide |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | transient immunosuppression |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | incidence |
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| Uncontrolled term | survival |
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| Uncontrolled term | genotype-phenotype correlations |
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| Uncontrolled term | health policies |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | CFTR modulators |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | human nasal epithelial cells |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | organoids |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | biomarker |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | functional assay |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | pre-clinical in vitro models |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | CFTR-related disorders |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | molecular diagnosis |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | CFTR variants |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | Next Generation Sequencing (NGS) |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | disease liability |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | interpretation |
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| Uncontrolled term | penetrance |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | genotype-guided therapy |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | miRNA |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | airway basal cell |
| 653 ## - INDEX TERM--UNCONTROLLED | |
| Uncontrolled term | lentivirus |
| 700 1# - ADDED ENTRY--PERSONAL NAME | |
| Personal name | Ferec, Claude |
| Relationship | edt |
| 700 1# - ADDED ENTRY--PERSONAL NAME | |
| Personal name | Yan, Ziying |
| Relationship | edt |
| 700 1# - ADDED ENTRY--PERSONAL NAME | |
| Personal name | Engelhardt, John |
| Relationship | oth |
| 700 1# - ADDED ENTRY--PERSONAL NAME | |
| Personal name | Ferec, Claude |
| Relationship | oth |
| 700 1# - ADDED ENTRY--PERSONAL NAME | |
| Personal name | Yan, Ziying |
| Relationship | oth |
| 856 40 - ELECTRONIC LOCATION AND ACCESS | |
| Host name | www.oapen.org |
| Uniform Resource Identifier | <a href="https://mdpi.com/books/pdfview/book/3205">https://mdpi.com/books/pdfview/book/3205</a> |
| Access status | 0 |
| Public note | DOAB: download the publication |
| 856 40 - ELECTRONIC LOCATION AND ACCESS | |
| Host name | www.oapen.org |
| Uniform Resource Identifier | <a href="https://directory.doabooks.org/handle/20.500.12854/69404">https://directory.doabooks.org/handle/20.500.12854/69404</a> |
| Access status | 0 |
| Public note | DOAB: description of the publication |
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