Drug development for gene therapy : translational biomarkers, bioanalysis, and companion diagnostics / edited by Yanmei Lu, Boris Gorovits
"This book focuses on the translational and bioanalytical challenges and best practices for gene therapy modalities. Some of the topics covered include study design, immunogenicity, various bioanalytical methods and their applications, and global regulatory issues. Altogether, the chapters offer an in-depth discussion of the basics and best practices for translational biomarkers, bioanalysis, and developing companion diagnostics / lab tests for gene therapies in the pharma and biopharma industry and research"--.
Introduction to AAV-based in vivo gene therapy -- Recent development in in vivo clinical gene therapy platforms -- Overview of biomarkers for AAV gene therapy -- Nonclinical and Clinical Study Considerations for Biodistribution, Shedding and Pharmacokinetics / Pharmacodynamics -- Immunogenicity of gene therapy products -- Bioanalytical Methods To Detect Pre-existing and Post-administration Humoral Immune Responses Against AAV Capsid Proteins -- Bioanalytical methods to study biodistribution and shedding of AAV-based gene therapy vectors -- Transgene mRNA expression analysis -- Quantification of transgene protein expression and biochemical function -- Substrate and Distal Pharmacodynamic Biomarker Measurements for Gene Therapy -- Detection of cellular immunity to viral capsids and transgene proteins -- Detection of humoral response to viral capsids and transgene proteins -- rAAV integration : Detection and Risk Assessment -- Gene modification and integration for patient monitoring -- Introduction to companion diagnostics for gene therapy -- Validation for Gene Therapy Companion Diagnostics -- Regulatory considerations for gene therapy companion diagnostics -- Current regulatory landscape for gene therapy product development and the role of biomarkers..
| Medienart: |
E-Book |
|---|
| Erscheinungsjahr: |
[2024] |
|---|---|
| Erschienen: |
Hoboken, New Jersey: Wiley ; 2024 |
| Weitere Ausgaben: |
Erscheint auch als Druck-Ausgabe: Drug development for gene therapy |
|---|
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Lu, Yanmei [HerausgeberIn] |
|---|
| Links: |
doi.org [lizenzpflichtig] |
|---|
| ISBN: |
|---|
| Nlm: |
QU 560 |
|---|---|
| Themen: |
| Anmerkungen: |
Includes bibliographical references and index |
|---|
| Umfang: |
1 Online-Ressource (xxiv, 465 Seiten) |
|---|
| doi: |
10.1002/9781119852810 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
1884377335 |
|---|
| LEADER | 01000cam a2200265 4500 | ||
|---|---|---|---|
| 001 | 1884377335 | ||
| 003 | DE-627 | ||
| 005 | 20240328151304.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 240326s2024 xxu|||||o 00| ||eng c | ||
| 020 | |a 9781119852810 |c obook |9 978-1-119-85281-0 | ||
| 020 | |a 9781119852797 |c adobe pdf |9 978-1-119-85279-7 | ||
| 020 | |a 9781119852803 |c epub |9 978-1-119-85280-3 | ||
| 024 | 7 | |a 10.1002/9781119852810 |2 doi | |
| 035 | |a (DE-627)1884377335 | ||
| 035 | |a (DE-599)KXP1884377335 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rda | ||
| 041 | |a eng | ||
| 044 | |c XD-US | ||
| 050 | 0 | |a RB155 | |
| 060 | 0 | |a QU 560 | |
| 082 | 0 | |a 616/.042 |2 23/eng/20231214 | |
| 245 | 1 | 0 | |a Drug development for gene therapy |b translational biomarkers, bioanalysis, and companion diagnostics |c edited by Yanmei Lu, Boris Gorovits |
| 264 | 1 | |a Hoboken, New Jersey |b Wiley |c [2024] | |
| 300 | |a 1 Online-Ressource (xxiv, 465 Seiten) | ||
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a Computermedien |b c |2 rdamedia | ||
| 338 | |a Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Includes bibliographical references and index | ||
| 520 | |a "This book focuses on the translational and bioanalytical challenges and best practices for gene therapy modalities. Some of the topics covered include study design, immunogenicity, various bioanalytical methods and their applications, and global regulatory issues. Altogether, the chapters offer an in-depth discussion of the basics and best practices for translational biomarkers, bioanalysis, and developing companion diagnostics / lab tests for gene therapies in the pharma and biopharma industry and research"-- | ||
| 520 | |a Introduction to AAV-based in vivo gene therapy -- Recent development in in vivo clinical gene therapy platforms -- Overview of biomarkers for AAV gene therapy -- Nonclinical and Clinical Study Considerations for Biodistribution, Shedding and Pharmacokinetics / Pharmacodynamics -- Immunogenicity of gene therapy products -- Bioanalytical Methods To Detect Pre-existing and Post-administration Humoral Immune Responses Against AAV Capsid Proteins -- Bioanalytical methods to study biodistribution and shedding of AAV-based gene therapy vectors -- Transgene mRNA expression analysis -- Quantification of transgene protein expression and biochemical function -- Substrate and Distal Pharmacodynamic Biomarker Measurements for Gene Therapy -- Detection of cellular immunity to viral capsids and transgene proteins -- Detection of humoral response to viral capsids and transgene proteins -- rAAV integration : Detection and Risk Assessment -- Gene modification and integration for patient monitoring -- Introduction to companion diagnostics for gene therapy -- Validation for Gene Therapy Companion Diagnostics -- Regulatory considerations for gene therapy companion diagnostics -- Current regulatory landscape for gene therapy product development and the role of biomarkers. | ||
| 650 | 2 | |a Genetic Therapy |x methods | |
| 650 | 2 | |a Biomarkers, Pharmacological |x analysis | |
| 650 | 2 | |a Drug Development |x methods | |
| 700 | 1 | |a Lu, Yanmei |e herausgeberin |4 edt | |
| 700 | 1 | |a Gorovits, Boris |e herausgeberin |4 edt | |
| 776 | 1 | |z 9781119852780 | |
| 776 | 0 | 8 | |i Erscheint auch als |n Druck-Ausgabe |t Drug development for gene therapy |d Hoboken, New Jersey : John Wiley & Sons, Inc, 2024 |h pages cm |w (DE-627)1876474793 |z 9781119852780 |
| 856 | 4 | 0 | |u https://doi.org/10.1002/9781119852810 |x Resolving-System |z lizenzpflichtig |
| 856 | 4 | 0 | |u https://onlinelibrary.wiley.com/doi/book/10.1002/9781119852810 |x Verlag |z lizenzpflichtig |
| 912 | |a ZDB-35-WIC | ||
| 912 | |a GBV_ILN_2011 | ||
| 912 | |a ISIL_DE-16 | ||
| 912 | |a SYSFLAG_1 | ||
| 912 | |a GBV_KXP | ||
| 951 | |a BO | ||
| 953 | |2 045F |a 616/.042 | ||
| 980 | |2 2011 |1 01 |x DE-16 |b 4504069032 |c 00 |f --%%-- |d --%%-- |e --%%-- |j knp |y l01 |z 26-03-24 | ||
| 981 | |2 2011 |1 01 |x DE-16 |r https://doi.org/10.1002/9781119852810 | ||