Autologous and cancer stem cell gene therapy / editors, Roger Bertolotti, Keiya Ozawa.

Format:

Book

Contributor:

Bertolotti, R. (Roger)

Ozawa, Keiya, 1953-

Series:

Progress in gene therapy ; v. 3.

Progress in gene therapy ; v. 3

Language:

English

Subjects (All):

Stem cells--Therapeutic use.

Stem cells.

Stem cells--Transplantation.

Gene therapy.

Cancer--Gene therapy.

Cancer.

Autotransplantation.

Physical Description:

1 online resource (488 p.)

Place of Publication:

Hackensack, NJ : World Scientific, c2008.

Language Note:

English

Summary:

Stem cells provide for life-long cell replacement in tissues and organs, and have inherent homing abilities that are critical in therapeutic applications. Stem cells are also the driving force of cancer where genetic/epigenetic alterations culminate in tumorigenesis either in tissue stem cells or in some of their derivatives. As a rare subset of the tumor, cancer stem cells are the only drive of tumor initiation/propagation. Autologous and cancer stem cells are thus the key targets of 1) long-term and transient-regenerative/epigenetic gene therapy and 2) of recurrence-free anticancer therapy,

Contents:

Contents; Preface Roger Bertolotti & Keiya Ozawa; List of Contributors; Introduction Toward a Universal Platform for Autologous Stem Cell Gene Therapy Roger Bertolotti; 1. Therapeutic Homing as the Very Drive of Both Long-term and Transient-Regenerative Stem Cell Gene Therapy; 2. Stem/Progenitor Cells as Tissue- or Tumor-homing Vectors for Targeted Transient Regenerative/Cancer Gene Therapy; 2.1. Autologous Stem/Progenitor Cells as Tissue-Homing Vectors forTargetedTransient Regenerative GeneTherapy

2.2. Autologous and Immortalized Stem/Progenitor Cells as Tumor-homing Vectors forTargeted Cancer GeneTherapy2.3. Cancer Stem Cells as Breakthrough Targets for Stem Cell-mediated GeneTherapy and Other CancerTherapies; 3. Therapeutic Homing and Autologous Stem Cells as the Main Drive of Our Stem Cell Gene Therapy Platform; 4. Autologous Pluripotent Stem Cells: Toward a Universal Stem Cell Gene Therapy Platform; 4.1. GeneTargeting and Cybridization as Genomic HomeostaticTools; 4.1.1. Endonuclease-boosted gene targeting; 4.1.2. Cybridization: transmitochondrial stem cells for mtDNA diseases

4.1.3. Genomic homeostasis and hazard-free custom site-specific integrative gene therapy4.2. Epigenetic Therapy: Potential Long-Term Gene Inactivation/ Activation Through Transient siRNA GeneTherapy; 4.3. Emerging Pluripotent Stem Cells as Potential Universal Drives for Tissue-Specific and Multisystemic GeneTherapy; References; Long-term Stem Cell Gene Therapy: From Current Clinical Random-Integration Achievements to Stem Cell Gene Targeting/Cybridization; I. From pioneering clinical retroviral gene therapy to experimental site-specific integrative gene therapy

1 Stem Cell Gene Therapy for ADA-Deficiency without Myelopreparative Conditioning Makoto Otsu and Tadashi Ariga1. Introduction; 2. Severe Combined Immunodeficiency due to ADA-deficiency; 3. Gene Therapy Trials for ADA-deficiency; 3.1. T Cell-Directed Gene Transfer as a Treatment for ADA-deficiency; 3.2. Early Stem Cell Gene Therapy Trials; 3.3. Second Generation of Stem Cell Gene Therapy Trials for ADA-deficiency; 4. Stem Cell Gene Therapy for ADA-deficiency without Myelopreparative Conditioning

5. Factors that Likely Affect the Safety and Efficacy of Stem Cell Gene Therapy for ADA-deficiency5.1. Age of Patients; 5.2. Transduction Procedures; 5.3. Cell Doses; 5.4. Use of PEG-ADA; 5.5. Cytoreductive Preconditioning; 6. Myelopreparative Treatment: Roles in Stem Cell Gene Therapy; 6.1. Concept of Myelopreparation; 6.2. Conditioning in SCGT: comparison between ADA-SCID and X-SCID; 7. Future of SCGT for ADA-deficiency; References

2 Targeted Insertion of Transgene into a Specific Site on Chromosome 19 by Using Adeno-Associated Virus Integration Machinery Masashi Urabe, Yoko Obara, Takayuki Ito, Hiroaki Mizukami, Akihiro Kume and Keiya Ozawa

Notes:

Description based upon print version of record.

Includes bibliographical references and index.

ISBN:

9786611928926

9781281928924

1281928925

9789812775870

9812775870

OCLC:

879023433