Lecture

Mod-09 Lec-33 Human Gene Therapy

This module focuses on human gene therapy, discussing its principles, methods, and potential applications in treating genetic disorders.

Key topics include:

  • Types of gene therapy approaches (e.g., somatic vs. germline)
  • Delivery methods for therapeutic genes
  • Case studies of successful gene therapy applications

Course Lectures
  • This module introduces eukaryotic RNA polymerases and the fundamental transcription factors required for initiating gene transcription. It discusses the structure and function of various polymerases, including RNA Pol I, II, and III, and their roles in synthesizing different RNA types.

    Key topics include:

    • Types of RNA polymerases and their specific functions
    • Basal transcription factors and their interactions with RNA polymerases
    • Initiation of transcription in eukaryotic cells
  • This module focuses on the diversity of core promoter elements in eukaryotic gene expression. It explores how different promoter sequences influence the binding of transcription machinery and the initiation of transcription.

    Key points include:

    • The role of core promoter elements in transcription initiation
    • Variability of promoter sequences across different genes
    • Interaction between promoter elements and transcription factors
  • This module delves into the diversity of general transcription factors essential for eukaryotic gene expression. It covers the various transcription factors, their roles, and how they interact with RNA polymerases to facilitate transcription.

    Topics include:

    • General transcription factor complexes and their components
    • How transcription factors recognize and bind to promoter regions
    • The importance of transcription factor diversity in regulating gene expression
  • This module examines proximal and distal promoter elements, enhancers, silencers, and gene-specific regulators. Understanding their functions is essential for grasping how eukaryotic gene expression is fine-tuned.

    Key areas include:

    • Definition and function of proximal versus distal elements
    • Role of enhancers and silencers in gene expression regulation
    • Mechanisms by which gene-specific regulators influence transcription
  • This module focuses on transcription factors' DNA binding domains, essential for recognizing and interacting with specific DNA sequences. The structure and function of these domains are crucial for understanding gene regulation.

    Key topics include:

    • Types of DNA binding domains
    • How these domains facilitate gene-specific transcriptional activation
    • Examples of transcription factors with distinct DNA binding motifs
  • This module addresses transcription factors' transcription activation domains, which are vital for recruiting the transcriptional machinery necessary for gene expression.

    Key areas covered include:

    • Definition and importance of transcription activation domains
    • How these domains interact with other proteins to start transcription
    • Examples of transcription factors with well-studied activation domains
  • This module investigates the role of chromatin in eukaryotic gene regulation. It explains how chromatin structure affects gene accessibility and transcriptional activity.

    Key topics include:

    • Chromatin structure and organization
    • Mechanisms by which chromatin remodeling influences transcription
    • Interaction between chromatin and transcription factors during gene activation
  • This module focuses on the role of histones in eukaryotic gene regulation. It discusses how histone modifications can influence gene expression by altering chromatin structure.

    Key areas include:

    • Histone proteins and their structural features
    • Types of histone modifications and their effects on transcription
    • How histone modifications are involved in epigenetic regulation
  • This module discusses the role of DNA methylation in eukaryotic gene regulation. It highlights how methylation patterns can influence gene expression and contribute to cellular differentiation.

    Key topics include:

    • Mechanisms of DNA methylation and demethylation
    • Impact of DNA methylation on gene accessibility and transcription
    • Examples of methylation's role in development and disease
  • This module examines chromatin remodeling and its implications for gene regulation. It discusses various chromatin remodeling complexes that alter chromatin structure to facilitate transcription.

    Key areas include:

    • Mechanisms of chromatin remodeling
    • Types of chromatin remodeling complexes
    • How remodeling affects gene expression and cellular function
  • This module focuses on the co-transcriptional and post-transcriptional modifications of pre-messenger RNA. It discusses how these modifications are critical for mRNA stability and translation.

    Key topics include:

    • The roles of capping, splicing, and polyadenylation in mRNA maturation
    • How these modifications influence gene expression
    • Examples of diseases associated with splicing defects
  • This module examines additional co-transcriptional and post-transcriptional modifications of pre-messenger RNA, focusing on the specific roles of RNA polymerase II in processing.

    Key areas include:

    • The importance of RNA capping and its role in mRNA stability
    • Mechanisms of mRNA splicing and its regulation
    • Polyadenylation and its effects on mRNA translation
  • This module focuses on the regulation of RNA Pol I transcription. It discusses how this polymerase is responsible for synthesizing ribosomal RNA (rRNA) and the factors that influence its activity.

    Key topics include:

    • Structure and function of RNA Pol I
    • Regulatory elements involved in rRNA synthesis
    • Factors impacting RNA Pol I transcription regulation
  • This module examines the regulation of tRNA and 5S rRNA synthesis by RNA Polymerase III. It highlights the significance of these RNA types in protein synthesis and cellular function.

    Key areas include:

    • Function and importance of tRNA and 5S rRNA
    • Regulatory mechanisms controlling RNA Polymerase III activity
    • Examples of diseases caused by dysregulation of tRNA synthesis
  • This module introduces signal transduction pathways, providing an overview of how extracellular signals are transmitted into cellular responses, leading to changes in gene expression.

    Key topics include:

    • Basic concepts of signal transduction
    • Types of signaling molecules and receptors
    • How signal transduction affects gene regulation
  • This module focuses on the regulation of gene expression by cyclic AMP (cAMP). It details how cAMP acts as a secondary messenger in signaling pathways that influence transcription.

    Key areas include:

    • Mechanism of cAMP signaling
    • Role of cAMP in activating protein kinases
    • Examples of genes regulated by cAMP pathways
  • This module discusses the regulation of gene expression by secondary messengers other than cAMP. It examines various signaling molecules and their roles in transcriptional regulation.

    Key topics include:

    • Types of secondary messengers and their functions
    • How these messengers influence transcription factors
    • Examples of pathways regulated by secondary messengers
  • This module focuses on the regulation of gene expression by Protein Kinase C (PKC). It discusses how PKC functions in various signaling pathways to modulate transcriptional activity.

    Key areas include:

    • Mechanism of PKC activation and signaling
    • Role of PKC in regulating transcription factors
    • Examples of genes affected by PKC-mediated pathways
  • This module examines the regulation of gene expression by growth factors. It explains how these signaling molecules influence cellular processes, including transcriptional regulation.

    Key topics include:

    • Types of growth factors and their roles
    • Mechanisms of growth factor signaling
    • Examples of genes regulated by growth factors
  • This module focuses on the regulation of gene expression by cytokines. It discusses how these immune signaling molecules affect transcription and cellular responses.

    Key areas include:

    • Types of cytokines and their functions
    • Mechanisms of cytokine signaling
    • Examples of genes regulated by cytokines
  • This module examines the regulation of gene expression by steroid hormones. It highlights the mechanisms by which these hormones influence transcriptional activity through intracellular receptors.

    Key topics include:

    • Mechanism of steroid hormone signaling
    • Role of steroid hormone receptors in transcription regulation
    • Examples of target genes regulated by steroid hormones
  • This module discusses the regulation of gene expression by type II nuclear receptors. It explains how these receptors function as transcription factors in response to steroid hormones and other ligands.

    Key areas include:

    • Structure and function of type II nuclear receptors
    • Mechanisms of ligand binding and transcriptional activation
    • Examples of genes regulated by type II nuclear receptors
  • This module focuses on the mechanism of transcriptional activation by nuclear receptors. It details how these receptors influence gene expression upon binding their ligands.

    Key topics include:

    • Mechanisms of transcriptional activation by nuclear receptors
    • Interaction with coregulators and transcription machinery
    • Examples of genes regulated by nuclear receptors
  • This module discusses gene regulation during Drosophila development, highlighting the mechanisms that control gene expression at various developmental stages.

    Key areas include:

    • Key genes involved in Drosophila development
    • Role of transcription factors and signaling pathways in development
    • Examples of gene regulatory networks in Drosophila
  • This module examines the signal transduction pathways involved in embryonic development, focusing on how these pathways regulate gene expression during early development.

    Key topics include:

    • Major signaling pathways and their roles in development
    • How signaling influences transcriptional regulation
    • Examples of developmental genes regulated by these pathways
  • Mod-07 Lec-26 Homeotic genes
    Prof. P.N. Rangarajan

    This module focuses on homeotic genes and their role in determining body plan and segment identity during development. It discusses how these genes are regulated.

    Key areas include:

    • Definition and function of homeotic genes
    • Regulatory mechanisms controlling homeotic gene expression
    • Examples of homeotic gene function in various organisms
  • This module discusses the epigenetic regulation of gene expression during development, focusing on how epigenetic modifications influence transcriptional activity.

    Key topics include:

    • Types of epigenetic modifications (e.g., methylation, histone modification)
    • How epigenetics affects gene expression during development
    • Examples of epigenetic changes linked to developmental processes
  • This module focuses on embryonic stem cells and their potential for transcription factor-mediated epigenetic reprogramming. It discusses how these cells can differentiate into various cell types.

    Key areas include:

    • Properties of embryonic stem cells
    • Mechanisms of transcription factor-mediated reprogramming
    • Applications of stem cells in regenerative medicine
  • This module discusses cloning and expression vectors, focusing on their design and application in molecular biology for gene cloning and protein expression.

    Key topics include:

    • Types of cloning vectors and their features
    • Expression vectors for protein production
    • Applications of vectors in research and biotechnology
  • This module focuses on eukaryotic protein expression systems, detailing different systems used to produce proteins in eukaryotic cells.

    Key areas include:

    • Overview of various eukaryotic expression systems
    • Advantages and disadvantages of each system
    • Applications of eukaryotic systems in research and industry
  • This module continues the discussion on eukaryotic protein expression systems, examining specific strategies for optimizing protein production in these systems.

    Key topics include:

    • Optimization techniques for eukaryotic expression systems
    • Factors affecting protein yield and quality
    • Success stories and examples of high-yield protein production
  • This module examines advanced eukaryotic protein expression systems, including the use of mammalian cells and viral vectors for gene expression.

    Key areas include:

    • Advantages of using mammalian cells for protein expression
    • Mechanisms of viral vector-mediated gene expression
    • Applications of these advanced systems in research and therapeutics
  • Mod-09 Lec-33 Human Gene Therapy
    Prof. P.N. Rangarajan

    This module focuses on human gene therapy, discussing its principles, methods, and potential applications in treating genetic disorders.

    Key topics include:

    • Types of gene therapy approaches (e.g., somatic vs. germline)
    • Delivery methods for therapeutic genes
    • Case studies of successful gene therapy applications
  • Mod-09 Lec-34 DNA vaccines
    Prof. P.N. Rangarajan

    This module discusses DNA vaccines, explaining their mechanism of action and potential as a novel approach in preventive medicine and immunotherapy.

    Key areas include:

    • How DNA vaccines elicit immune responses
    • Advantages of DNA vaccines over traditional vaccines
    • Examples of DNA vaccines in clinical trials
  • Mod-09 Lec-35 Transgenic animals
    Prof. P.N. Rangarajan

    This module focuses on transgenic animals and their significance in research and biotechnology, discussing methods for creating and utilizing these organisms.

    Key topics include:

    • Methods for generating transgenic animals
    • Applications of transgenic animals in biomedical research
    • Ethical considerations surrounding the use of transgenic animals
  • Mod-09 Lec-36 Transgenic plants
    Prof. P.N. Rangarajan

    This module discusses transgenic plants, focusing on the methods used to create these organisms and their applications in agriculture and environmental science.

    Key areas include:

    • Techniques for generating transgenic plants
    • Benefits of transgenic plants in agriculture
    • Environmental applications of transgenic plants
  • Mod-09 Lec-37 Knockout mice
    Prof. P.N. Rangarajan

    This module focuses on knockout mice, discussing how these genetically modified organisms are created and their applications in studying gene function.

    Key topics include:

    • Techniques for generating knockout mice
    • Applications of knockout mice in biomedical research
    • Implications for understanding genetic diseases
  • This module discusses the regulation of eukaryotic gene expression by small RNAs, particularly RNA interference (RNAi). It covers the mechanisms by which small RNAs influence gene regulation.

    Key areas include:

    • Mechanisms of RNA interference and small RNA biogenesis
    • Roles of small RNAs in gene silencing
    • Applications of RNAi in research and therapeutic interventions
  • This module focuses on genomics and proteomics, discussing how high-throughput techniques are used to study gene expression and protein function on a large scale.

    Key topics include:

    • Genomic technologies for analyzing gene expression
    • Proteomic approaches for studying protein interactions
    • Applications of genomics and proteomics in research and medicine
  • This module discusses metabolic engineering and synthetic biology, focusing on how these fields are applied to modify and design biological systems for various applications.

    Key areas include:

    • Principles of metabolic engineering
    • Applications of synthetic biology in biotechnology
    • Future directions and challenges in these fields