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Keynote Essay 6: Molecular Research Case Study: Developing Novel RNA Interference-based Therapy
- from SECTION 3 - MOLECULAR THERAPEUTICS
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- By Patrick Arbuthnot, MB BCh, BSc (Hons), PhD, is a reader in the Division of Molecular Medicine and Haematology at the Medical School of the University of the Witwatersrand, Johannesburg. He has a research interest in using nucleic acid transfer to develop new approaches to treating viral infections of South African importance., Marc S Weinberg, PhD, is a senior lecturer in the Division of Molecular Medicine and Haematology, University of the Witwatersrand, and a member of the Antiviral Gene Therapy Research Unit.
- Edited by Barry Mendelow, University of the Witwatersrand, Johannesburg, Michèle Ramsay, University of the Witwatersrand, Johannesburg, Nanthakumarn Chetty, University of the Witwatersrand, Johannesburg, Wendy Stevens, University of the Witwatersrand, Johannesburg
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- Book:
- Molecular Medicine for Clinicians
- Published by:
- Wits University Press
- Published online:
- 04 June 2019
- Print publication:
- 01 October 2008, pp 441-448
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Summary
THE VALUE OF ACADEMIC RESEARCH
Research can be defined as the process of discovery and creation of new knowledge. A vital role for universities is to carry out research, and success in this activity is a good gauge of the well-being of these academic institutions. All leading universities of the world (such as Harvard, Cambridge and Oxford universities) have a strong commitment to supporting research, and this is the foundation of their good reputations. Research is vital to any society and positive outcomes go way beyond the direct practical application of new discoveries. By nurturing creativity, critical thinking, open-mindedness and enquiry, research has the broad benefits of building a strong democratic ethic in society. Today we refer to the importance of the ‘knowledge based economy’ where societies become more reliant on creation and exploitation of new knowledge to understand and overcome economic and political challenges. An active research community allows countries to benefit directly from new developments of the twenty first century and to participate in developing a knowledge-based economy. The benefits of an active research environment can have both direct and indirect effects on society. Some of these benefits are listed below.
• Improved training of students. An often under-appreciated role of research is that it is beneficial to teaching in universities. By and large, research activities are supportive of university educational programmes, and teachers who are immersed in active research generally make better instructors. Research leads to more meaningful insight into a subject, and educators pass this benefit on to students through better teaching. High-level training of students that is not based on a foundation of academic research risks becoming stale and outmoded.
• Promoting innovation for patient care. Insights gained from research on medical challenges contribute significantly to understanding and overcoming patient problems, which collectively makes for better patient management and care.
• Broadening the knowledge base for the good of humanity. Some of the most significant breakthroughs in technology have come from unlikely research contributions. Research does not always produce immediately obvious practical benefits, and it is often only after several years that the utility of research becomes apparent.
Chapter 3 - The Anatomy and Physiology of the Genome
- from Section 1 - Principles Of Cellular And Molecular Biology
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- By Marc S Weinberg, PhD, is a senior lecturer in the Division of Molecular Medicine and Haematology, University of the Witwatersrand, and a member of the Antiviral Gene Therapy Research Unit., Natalie A Whalley, PhD, lectures in the Division of Molecular Medicine and Haematology, School of Pathology, University of the Witwatersrand., Michèle Ramsay, PhD (Human Genetics), is currently Professor and Head of the Molecular Genetics Laboratory, Division of Human Genetics, National Health Laboratory Service and University of the Witwatersrand.
- Edited by Barry Mendelow, University of the Witwatersrand, Johannesburg, Michèle Ramsay, University of the Witwatersrand, Johannesburg, Nanthakumarn Chetty, University of the Witwatersrand, Johannesburg, Wendy Stevens, University of the Witwatersrand, Johannesburg
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- Book:
- Molecular Medicine for Clinicians
- Published by:
- Wits University Press
- Published online:
- 04 June 2019
- Print publication:
- 01 October 2008, pp 19-36
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Summary
INTRODUCTION
The genome represents the entire genetic complement of an organism and is a repository of biological information, which is used to create and sustain every living system. Typically, a genome is composed of nucleic acids, with deoxyribonucleic acid or DNA being the most common form, although some viral genomes are composed of ribonucleic acid or RNA. DNA is a polymeric chain defined by a sequence of monomeric units called nucleotides. The asymmetrical arrangement of the nucleotide sequence of DNA or RNA represents a ‘code’ that defines the functional and structural role of a genome within an organism. It is therefore not surprising that the genome is frequently referred to as the ‘blueprint of life’.
Although all living organisms contain genomes, the focus of this chapter will be on the human genome, which is composed of two distinct sections: nuclear and mitochondrial. The nuclear genome is by far the largest section and comprises about 3.2 billion nucleotides, which are divided into 24 linear molecules of DNA arranged into structures called chromosomes. The mitochondrial genome is a much smaller circular molecule of DNA comprising 16 569 nucleotides. Many mitochondrial organelles are found within a cell, allowing for multiple (approximately 8000) copies of this genome to be present.
Mere numbers can often deceive one, since everything operates at the molecular level. This molecular scale needs to be appreciated better. For example, there are over 100 000 000 000 000 cells in a typical adult human, and most cells have two complete copies (diploid) of the nuclear genome. If one were to convert the sequence of nucleotides into alphabetical letters this would equate to approximately 3000 volumes of Gray's Anatomy per cell! The past two decades have seen the completion of the sequencing of the human genome, a monu - mental scientific feat. However, we are now faced with the challenge of deciphering this code, a task that brings biology and medicine into the realm of the information sciences. This phenomenon is not dissimilar to the evolution of the computer that led to the development of information technology and the modern digital revolution.
Chapter 36 - Gene Therapy
- from SECTION 3 - MOLECULAR THERAPEUTICS
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- By Marc S Weinberg, PhD, is a senior lecturer in the Division of Molecular Medicine and Haematology, University of the Witwatersrand, and a member of the Antiviral Gene Therapy Research Unit., Patrick Arbuthnot, MB BCh, BSc (Hons), PhD, is a reader in the Division of Molecular Medicine and Haematology at the Medical School of the University of the Witwatersrand, Johannesburg. He has a research interest in using nucleic acid transfer to develop new approaches to treating viral infections of South African importance.
- Edited by Barry Mendelow, University of the Witwatersrand, Johannesburg, Michèle Ramsay, University of the Witwatersrand, Johannesburg, Nanthakumarn Chetty, University of the Witwatersrand, Johannesburg, Wendy Stevens, University of the Witwatersrand, Johannesburg
-
- Book:
- Molecular Medicine for Clinicians
- Published by:
- Wits University Press
- Published online:
- 04 June 2019
- Print publication:
- 01 October 2008, pp 413-421
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Summary
INTRODUCTION
It is fair to say that almost all diseases, whether acquired or inherited, have at their foundation a genetic aetiology. The understanding of the molecular basis of disease and the recent acquisition of ‘genetic’ tools have made it feasible to consider the notion of repairing damaged genes (e.g. inherited diseases) or removing rogue genetic elements (e.g. cancers, viruses and bacteria) as a viable therapeutic objective. But what is gene therapy? Although definitions may vary, in essence gene therapy refers to any procedure intended to treat or alleviate disease by genetically modifying the cells of a patient. Often, although not always, gene therapy involves the artificial introduction of genetic material (such as DNA or RNA) into cells. Since DNA and RNA are relatively large nucleic acid macromolecules, their successful introduction rests on developing delivery systems that are capable of carrying macro - molecules to diseased cells.
At present gene therapy remains an experimental technique with tremendous promise for the future of medicine. In this chapter different types of gene-based therapeutic approaches are outlined, with the focus being on innovative experimental technologies that are most likely to obtain clinical success. The topic of gene delivery will be discussed by comparing the strengths and weaknesses of viral and non-viral delivery systems
SOMATIC VS GERMLINE GENE THERAPY
Germline gene therapy. This form of gene therapy requires the introduction of genetic material (DNA or RNA) into sperm or oocytes to produce permanent, transmissible modification. Germline gene therapy is considered to be unethical and in some countries is illegal. In addition, this type of intervention may not necessarily solve the problem. For example, for many recessive disorders, carriers maintain the frequency of the dominant allele.
Somatic gene therapy. All current gene therapy treatment modalities aim to introduce genetic material into somatic cells, although, unlike germline therapy, such an approach is unlikely to produce permanent modifications of the targeted cells. Long-term trans mission of genes can be achieved by utilising viral vectors capable of integrating into quiescent (non-dividing) cells or by targeting stem cells.
Defect-Induced Shifts in the Elastic Constants of Silicon
- Clark L. Allred, Jeffrey T. Borenstein, Marc S. Weinberg, Xianglong Yuan, Martin Z. Bazant, Linn W. Hobbs
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- Journal:
- MRS Online Proceedings Library Archive / Volume 741 / 2002
- Published online by Cambridge University Press:
- 11 February 2011, J5.26
- Print publication:
- 2002
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As MEMS devices become ever more sensitive, even slight shifts in materials properties can be detrimental to device performance. Radiation-induced defects can change both the dimensions and mechanical properties of MEMS materials, which will be of concern to designers of MEMS for applications involving radiation exposure, such as those in a reactor environment or in space. We have performed atomistic simulations of the effect that defects and amorphous regions, such as could be produced by radiation damage, have on the elastic constants of silicon. We have then applied the results of the elastic constant shift calculations to a hypothetical MEMS device, and calculated the difference that would be generated by this effect.