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Genetic modification of hematopoietic stem cells methods and protocols 1st Edition by Guillermo Guenechea, Jose C Segovia, Christopher Baum ISBN 1597454095 9781597454094

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Genetic modification of hematopoietic stem cells methods and protocols 1st Edition Guillermo Guenechea Digital Instant Download

Author(s): Guillermo Guenechea, Jose C. Segovia (auth.), Christopher Baum (eds.)
ISBN(s): 9781597454094, 1597454095
Edition: 1
File Details: PDF, 8.03 MB
Year: 2009
Language: english
SKU: EB-1222350 Category: Tags: , ,

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ISBN 10: 1597454095 
ISBN 13: 9781597454094
Author: Guillermo Guenechea, Jose C. Segovia, Christopher Baum

Gene Transfer into Hematopoietic Cells: From Basic Science to Clinical Application Christopher Baum 1. The Potential of Gene Transfer into Hematopoietic Vectors with the potential for stable transgene integration are Cells widely used in basic hematology and clinical trials of gene me- cine. In basic research, both gain-of-function and loss-of-fu- tion situations of individual genes can be created by gene transfer, leading to a wide range of applications in developmental biology, stem cell biology, immunology, leukemia research, and human genetics. With the first evidence of successful modification of murine hematopoietic cells using retroviral gene vectors (1, 2), researchers have also explored the therapeutic potential of this approach. To date, the emerging discipline of gene therapy is a highly diversified field that offers entirely novel approaches to treat a great variety of human diseases (3). All hematopoietic cell types are of major interest in this context, since the modification of the hematopoietic stem cell population may potentially give rise to a completely transgenic hematopoiesis with the potential to cure genetic disorders or fight severe chronic infections, and the targeting of mature cells such as lymphocytes or antigen-p- senting dendritic cells offers all types of transient and semiper- nent modifications of the immune system. The unifying principle of gene medicine is the need to transfer complex nucleic acids cells that do not contribute to the germline (somatic cells).

Genetic modification of hematopoietic stem cells methods and protocols 1st Table of contents:

Basics of Hematopoietic Stem Cell Biology

  • Definition and Role of HSCs in the Immune and Hematopoietic Systems

  • Properties of HSCs: Self-Renewal, Differentiation, and Engraftment

  • Niche and Microenvironmental Factors Influencing HSCs

  • HSC Isolation and Characterization

3. Methods for Genetic Modification of Hematopoietic Stem Cells

  • Overview of Genetic Modification Techniques

  • Classical Methods: Transfection, Transduction, and Electroporation

  • Viral Vectors: Lentivirus, Retrovirus, Adenovirus, and AAV

  • Non-Viral Methods: CRISPR/Cas9, TALENs, ZFNs, and mRNA-based Approaches

  • Genomic Integration vs. Episomal Maintenance

4. Preparation and Isolation of Hematopoietic Stem Cells

  • Collection of HSCs from Peripheral Blood, Bone Marrow, and Umbilical Cord Blood

  • Enrichment Strategies for Isolating HSCs: Positive vs. Negative Selection

  • Flow Cytometry-Based Isolation of HSCs

  • Quality Control and Characterization of Isolated HSCs

5. Gene Editing Technologies: Protocols and Applications

  • CRISPR/Cas9 Technology for HSC Genome Editing

  • TALENs (Transcription Activator-Like Effector Nucleases)

  • ZFNs (Zinc Finger Nucleases)

  • Base and Prime Editing: Next-Generation Genome Editing

  • Optimizing Delivery of Editing Components into HSCs

6. Protocols for Transfection and Transduction of HSCs

  • Electroporation Protocols for Efficient DNA Delivery

  • Lentiviral and Retroviral Transduction Methods

  • Optimizing Viral Titers for High Efficiency

  • Protocols for mRNA and CRISPR-Cas9 Delivery

  • Screening and Selection of Transduced or Transfected Cells

7. In Vitro Expansion of Modified Hematopoietic Stem Cells

  • Media Formulations for HSC Expansion

  • Growth Factors and Cytokines to Support HSC Proliferation

  • Maintenance of Stemness During Expansion

  • Monitoring Cell Viability, Growth, and Differentiation

8. In Vivo Transplantation and Engraftment Protocols

  • Preclinical Models for Testing Modified HSCs (Murine, Xenograft Models)

  • In Vivo Transplantation of Modified HSCs into Recipient Mice

  • Evaluation of Engraftment and Long-Term Reconstitution

  • Assessing Hematopoietic Lineage Development Post-Transplantation

9. Assessing the Functional Outcomes of Genetic Modification

  • Hematopoietic Reconstitution and Blood Cell Lineage Analysis

  • Flow Cytometry for Identifying Differentiated Lineages

  • Functional Assays for Immune Reconstitution (e.g., T-cell, B-cell)

  • In Vivo Monitoring of Gene Expression and Cell Fate

10. Safety and Quality Control in Genetic Modification of HSCs

  • Assessing Off-Target Effects in Genome Editing

  • Mitigation Strategies for Ensuring Precision in Gene Editing

  • Validation of Gene Modification by Sequencing, qPCR, and Western Blot

  • Contamination and Cross-Contamination Risks in Cell Culture

11. Applications of Genetically Modified Hematopoietic Stem Cells

  • Gene Therapy for Hematological Disorders (e.g., Sickle Cell Anemia, Thalassemia)

  • Immunotherapy Applications: CAR-T Cells and HSC Engineering for Cancer

  • Engineering HSCs for HIV Resistance or Immunodeficiency Treatment

  • Potential for Hematopoietic Stem Cell-Based Regenerative Medicine

12. Current Challenges and Future Directions

  • Limitation of Current Genetic Modification Methods

  • Challenges in Transgene Stability and Long-Term Engraftment

  • Overcoming Immunogenicity in Transplanted Modified HSCs

  • The Role of Artificial Intelligence and Computational Modeling in Gene Editing

  • Next-Generation Technologies: CRISPR Prime Editing, Epigenetic Modifications

13. Ethical, Regulatory, and Clinical Considerations

  • Ethical Considerations in Gene Editing of HSCs

  • Regulatory Framework for Clinical Trials Involving Gene-Modified HSCs

  • Translating Preclinical Research to Clinical Applications

  • Public Perception and Ethical Challenges in Genetic Modifications

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Tags: Guillermo Guenechea, Jose C Segovia, Christopher Baum, Genetic modification, hematopoietic stem cells