Concepts in genetic medicine:
Gespeichert in:
| Format: | Buch |
|---|---|
| Sprache: | English |
| Veröffentlicht: |
Hoboken, NJ [u.a.]
Wiley-Liss
2008
|
| Schlagworte: | |
| Online-Zugang: | Table of contents only Publisher description Klappentext Inhaltsverzeichnis |
| Beschreibung: | XIII, 377 S. Ill., graph. Darst. |
| ISBN: | 9780471703204 0471703206 |
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| 650 | 4 | |a Medical genetics | |
| 650 | 4 | |a Gene therapy | |
| 650 | 4 | |a Gene Therapy |x methods |v Review | |
| 650 | 4 | |a Gene Transfer Techniques |v Review | |
| 650 | 4 | |a Genetic Vectors |v Review | |
| 650 | 4 | |a Viruses |x genetics |v Review | |
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Datensatz im Suchindex
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| adam_text | While the field of gene therapy is still a relatively young discipline, it is rapidly
evolving and is characterized by impressive scientific and medical innovation,
¡n Concepts in Genetic Medicine, readers are offered a collection of uniformly
organized and succinct reviews in the field of therapeutic genetic medicine
presented by a carefully selected group of top experts. Each chapter focuses
on a single, current topic and explains issues in the development of genetic
treatments, critical challenges and strategies for implementing them, and
future directions in research and translational applications.
Beginning with an introduction to the genetic basis of disease and a discussion
of specific diseases, their etiology, and promising therapeutic treatments,
the book goes on to cover vector design, genetic payioads and disease targets,
vaccine vector systems, manufacture, assays, cell systems and target issues,
predinical testing, regulatory considerations, clinical evaluation, safety
concerns, and commercialization.
Complete with uniformly designed illustrations and a supplementary Web
site with a PowerPoint presentation for educators, this is a practical guide for
physicians, scientists, allied health workers, and patient advocacy professionals
attempting to develop genetic medicines for translation into the clinic.
RIBUTORS IX PREFACE XIIL INTRODUCTION 1 THE EVOLUTION OF HUMAN GENE
THERAPY: A JOURNEY FROM EXCESSIVE HYPE TO EXCESSIVE DIFFIDENCE TO
REALITY 1 THEODORE FRIEDMANN VECTOR SYSTEMS 2 MURINE LEUKEMIA
VIRUS-BASED RETROVIRAL VECTORS 7 KENNETH CORNETTA 3 LENTLVIRUS VECTORS
19 GWENDOLYN K. BINDER AND BORA DROPULIC 4 ADENOVIRAL VECTORS: HISTORY
AND PERSPECTIVE 39 DOUGLAS J. JOLLY, ESTUARDO AGUILAR-CORDOVA, AND LAURA
K. AGUILAR 5 ADENO-ASSOCIATED VIRUS VECTORS 61 BARRIE J. CARTER 6 SV40
VIRUS-DERIVED VECTORS 69 DAVID S. STRAYER 7 HERPES SIMPLEX VIRUS VECTORS
85 ANNA-MARIA ANESTI AND ROBERT S. COFFIN 8 NOEVIRAL GENE DELIVERY
SYSTEMS TAKESHI SUDA AND DEXI LIU VI CONTENTS 9 THERAPEUTIC GENE
TRANSFER TO SKELETAL MUSCLE 123 PAUL GREGOREVIC AND JEFFREY S.
CHAMBERLAIN 10 GENE THERAPY FOR CARDIOVASCULAR DISEASE 129 CHRISTINA A.
PACAK, KERRY O. CRESAWN, AND BARRY J. BYRNE 11 INTRAARTICULAR VECTOR
DELIVERY FOR INFLAMMATORY JOINT DISEASE 137 HAIM BURSTEIN 12 THE
RESPIRATORY SYSTEM AS A PLATFORM FOR GENE DELIVERY 145 BARRIE J. CARTER
13 THE BRAIN AS A TARGET FOR GENE THERAPY 153 PEDRO R. LOWENSTEIN AND
MARIA G. CASTRO 14 IMMUNE RESPONSES TO VIRAL VECTORS INJECTED
SYSTEMICALLY OR INTO THE CNS 167 PEDRO R. LOWENSTEIN AND MARIA G.CASTRO
15 CANCER VACCINES 181 RIMAS J. ORENTAS 16 GENETICALLY MODIFIED T CELLS
FOR HUMAN GENE THERAPY 193 JAMES L. RILEY AND CARL H. JUNE 17 LENTIVIRAL
VECTOR DELIVERY OF RNAI FOR THE TREATMENT OF HIV-1 INFECTION 207 KEVIN
V. MORRIS AND JOHN J. ROSSI 18 VECTOR TARGETING 223 QIANA L. MATTHEWS
AND DAVID T. CURIEL THE MANUFACTURE OF GENE THERAPY PRODUCTS 19 THE
MANUFACTURE OF GENETIC VIRAL VECTOR PRODUCTS 229 DOUGLAS J. JOLLY AND
ESTUARDO AGUILAR-CORDOVA 20 THE MANUFACTURE OF ADEN-ASSOCIATED VIRAL
VECTORS 245 RICHARD PELUSO CONTENTS VII 21 LENTI VIRUS VECTOR
MANUFACTURING 253 GWENDOLYN K. BINDER AND BORO DROPULIC SAFETY AND
REGULATORY CONSIDERATIONS 22 ASSAYS FOR THE RELEASE OF VIRAL VECTOR GENE
THERAPY PRODUCTS FOR HUMAN CLINICAL TRIALS 269 FLAVIA BORELLINI 23
SAFETY OF RETROVIRAI VECTORS: REGULATORY AND TECHNICAL CONSIDERATIONS
277 KENNETH CORNETTA AND CAROLYN A. WILSON 24 ASSAYS FOR THE QUALITY
CONTROL OF LENTIVIRAL VECTORS 289 JAMES E. MISKIN, SUSAN M. KINGSMAN,
AND KYRIACOS A. MITROPHANOUS 25 ASSAYS FOR NONVIRAL VECTORS 299 RALPH W.
PAUL 26 ASSAYS FOR THE RELEASE OF CELLULAR GENE THERAPY PRODUCTS 3CI7
PHILIP J. CROSS AND BRUCE L. LEVINE 27 TOXICOLOGY FOR GENE THERAPY
PRODUCTS: CONCEPTS AND CHALLENGES 319 RUSSETTE M. LYONS 28 REGULATORY
ASPECTS OF GENE THERAPY MEDICINAL PRODUCTS IN THE EUROPEAN UNION 329
MATTHIAS SCHWEIZER, CHRISTIAN J. BUCHHOLZ, AND KLAUS CICHUTEK FINANCE
AND REIMBURSMENT 29 VENTURE CAPITAL AND BIOTECHNOLOGY STARTUPS 341
DOUGLAS D. LIND 30 THE ROLE OF INVESTMENT BANKING IN THE GROWTH OF
BIOTECHNOLOGY COMPANIES 351 DOUGLAS J. SWIRSKY 31 MANAGING REIMBURSEMENT
FOR GENE THERAPY PRODUCTS 355 MARTIN GOLD INDEX 361
|
| adam_txt |
While the field of gene therapy is still a relatively young discipline, it is rapidly
evolving and is characterized by impressive scientific and medical innovation,
¡n Concepts in Genetic Medicine, readers are offered a collection of uniformly
organized and succinct reviews in the field of therapeutic genetic medicine
presented by a carefully selected group of top experts. Each chapter focuses
on a single, current topic and explains issues in the development of genetic
treatments, critical challenges and strategies for implementing them, and
future directions in research and translational applications.
Beginning with an introduction to the genetic basis of disease and a discussion
of specific diseases, their etiology, and promising therapeutic treatments,
the book goes on to cover vector design, genetic payioads and disease targets,
vaccine vector systems, manufacture, assays, cell systems and target issues,
predinical testing, regulatory considerations, clinical evaluation, safety
concerns, and commercialization.
Complete with uniformly designed illustrations and a supplementary Web
site with a PowerPoint presentation for educators, this is a practical guide for
physicians, scientists, allied health workers, and patient advocacy professionals
attempting to develop genetic medicines for translation into the clinic.
'RIBUTORS IX PREFACE XIIL INTRODUCTION 1 THE EVOLUTION OF HUMAN GENE
THERAPY: A JOURNEY FROM EXCESSIVE HYPE TO EXCESSIVE DIFFIDENCE TO
REALITY 1 THEODORE FRIEDMANN VECTOR SYSTEMS 2 MURINE LEUKEMIA
VIRUS-BASED RETROVIRAL VECTORS 7 KENNETH CORNETTA 3 LENTLVIRUS VECTORS
19 GWENDOLYN K. BINDER AND BORA DROPULIC 4 ADENOVIRAL VECTORS: HISTORY
AND PERSPECTIVE 39 DOUGLAS J. JOLLY, ESTUARDO AGUILAR-CORDOVA, AND LAURA
K. AGUILAR 5 ADENO-ASSOCIATED VIRUS VECTORS 61 BARRIE J. CARTER 6 SV40
VIRUS-DERIVED VECTORS 69 DAVID S. STRAYER 7 HERPES SIMPLEX VIRUS VECTORS
85 ANNA-MARIA ANESTI AND ROBERT S. COFFIN 8 NOEVIRAL GENE DELIVERY
SYSTEMS TAKESHI SUDA AND DEXI LIU VI CONTENTS 9 THERAPEUTIC GENE
TRANSFER TO SKELETAL MUSCLE 123 PAUL GREGOREVIC AND JEFFREY S.
CHAMBERLAIN 10 GENE THERAPY FOR CARDIOVASCULAR DISEASE 129 CHRISTINA A.
PACAK, KERRY O. CRESAWN, AND BARRY J. BYRNE 11 INTRAARTICULAR VECTOR
DELIVERY FOR INFLAMMATORY JOINT DISEASE 137 HAIM BURSTEIN 12 THE
RESPIRATORY SYSTEM AS A PLATFORM FOR GENE DELIVERY 145 BARRIE J. CARTER
13 THE BRAIN AS A TARGET FOR GENE THERAPY 153 PEDRO R. LOWENSTEIN AND
MARIA G. CASTRO 14 IMMUNE RESPONSES TO VIRAL VECTORS INJECTED
SYSTEMICALLY OR INTO THE CNS 167 PEDRO R. LOWENSTEIN AND MARIA G.CASTRO
15 CANCER VACCINES 181 RIMAS J. ORENTAS 16 GENETICALLY MODIFIED T CELLS
FOR HUMAN GENE THERAPY 193 JAMES L. RILEY AND CARL H. JUNE 17 LENTIVIRAL
VECTOR DELIVERY OF RNAI FOR THE TREATMENT OF HIV-1 INFECTION 207 KEVIN
V. MORRIS AND JOHN J. ROSSI 18 VECTOR TARGETING 223 QIANA L. MATTHEWS
AND DAVID T. CURIEL THE MANUFACTURE OF GENE THERAPY PRODUCTS 19 THE
MANUFACTURE OF GENETIC VIRAL VECTOR PRODUCTS 229 DOUGLAS J. JOLLY AND
ESTUARDO AGUILAR-CORDOVA 20 THE MANUFACTURE OF ADEN-ASSOCIATED VIRAL
VECTORS 245 RICHARD PELUSO CONTENTS VII 21 LENTI VIRUS VECTOR
MANUFACTURING 253 GWENDOLYN K. BINDER AND BORO DROPULIC SAFETY AND
REGULATORY CONSIDERATIONS 22 ASSAYS FOR THE RELEASE OF VIRAL VECTOR GENE
THERAPY PRODUCTS FOR HUMAN CLINICAL TRIALS 269 FLAVIA BORELLINI 23
SAFETY OF RETROVIRAI VECTORS: REGULATORY AND TECHNICAL CONSIDERATIONS
277 KENNETH CORNETTA AND CAROLYN A. WILSON 24 ASSAYS FOR THE QUALITY
CONTROL OF LENTIVIRAL VECTORS 289 JAMES E. MISKIN, SUSAN M. KINGSMAN,
AND KYRIACOS A. MITROPHANOUS 25 ASSAYS FOR NONVIRAL VECTORS 299 RALPH W.
PAUL 26 ASSAYS FOR THE RELEASE OF CELLULAR GENE THERAPY PRODUCTS 3CI7
PHILIP J. CROSS AND BRUCE L. LEVINE 27 TOXICOLOGY FOR GENE THERAPY
PRODUCTS: CONCEPTS AND CHALLENGES 319 RUSSETTE M. LYONS 28 REGULATORY
ASPECTS OF GENE THERAPY MEDICINAL PRODUCTS IN THE EUROPEAN UNION 329
MATTHIAS SCHWEIZER, CHRISTIAN J. BUCHHOLZ, AND KLAUS CICHUTEK FINANCE
AND REIMBURSMENT 29 VENTURE CAPITAL AND BIOTECHNOLOGY STARTUPS 341
DOUGLAS D. LIND 30 THE ROLE OF INVESTMENT BANKING IN THE GROWTH OF
BIOTECHNOLOGY COMPANIES 351 DOUGLAS J. SWIRSKY 31 MANAGING REIMBURSEMENT
FOR GENE THERAPY PRODUCTS 355 MARTIN GOLD INDEX 361 |
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| index_date | 2024-07-02T18:59:24Z |
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| language | English |
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| spelling | Concepts in genetic medicine ed. by Boro Dropulic ... Genetic medicine Hoboken, NJ [u.a.] Wiley-Liss 2008 XIII, 377 S. Ill., graph. Darst. txt rdacontent n rdamedia nc rdacarrier Medical genetics Gene therapy Gene Therapy methods Review Gene Transfer Techniques Review Genetic Vectors Review Viruses genetics Review Humangenetik (DE-588)4072653-8 gnd rswk-swf Gentherapie (DE-588)4296957-8 gnd rswk-swf Humangenetik (DE-588)4072653-8 s Gentherapie (DE-588)4296957-8 s b DE-604 Dropulic, Boro Sonstige oth http://www.loc.gov/catdir/toc/ecip0712/2007008049.html Table of contents only http://www.loc.gov/catdir/enhancements/fy0740/2007008049-d.html Publisher description Digitalisierung UB Regensburg application/pdf http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=016149475&sequence=000003&line_number=0001&func_code=DB_RECORDS&service_type=MEDIA Klappentext SWB Datenaustausch application/pdf http://bvbr.bib-bvb.de:8991/F?func=service&doc_library=BVB01&local_base=BVB01&doc_number=016149475&sequence=000004&line_number=0002&func_code=DB_RECORDS&service_type=MEDIA Inhaltsverzeichnis |
| spellingShingle | Concepts in genetic medicine Medical genetics Gene therapy Gene Therapy methods Review Gene Transfer Techniques Review Genetic Vectors Review Viruses genetics Review Humangenetik (DE-588)4072653-8 gnd Gentherapie (DE-588)4296957-8 gnd |
| subject_GND | (DE-588)4072653-8 (DE-588)4296957-8 |
| title | Concepts in genetic medicine |
| title_alt | Genetic medicine |
| title_auth | Concepts in genetic medicine |
| title_exact_search | Concepts in genetic medicine |
| title_exact_search_txtP | Concepts in genetic medicine |
| title_full | Concepts in genetic medicine ed. by Boro Dropulic ... |
| title_fullStr | Concepts in genetic medicine ed. by Boro Dropulic ... |
| title_full_unstemmed | Concepts in genetic medicine ed. by Boro Dropulic ... |
| title_short | Concepts in genetic medicine |
| title_sort | concepts in genetic medicine |
| topic | Medical genetics Gene therapy Gene Therapy methods Review Gene Transfer Techniques Review Genetic Vectors Review Viruses genetics Review Humangenetik (DE-588)4072653-8 gnd Gentherapie (DE-588)4296957-8 gnd |
| topic_facet | Medical genetics Gene therapy Gene Therapy methods Review Gene Transfer Techniques Review Genetic Vectors Review Viruses genetics Review Humangenetik Gentherapie |
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