Genes and Cell Therapy Analysis Essay examples

Genes and Cell Therapy Analysis Essay examples

Length: 2703 words (7.7 double-spaced pages)

Rating: Research Papers

Open Document

Essay Preview

The article selected for this assignment is “Targeted Gene Correction of α1-antitrypsin Deficiency in Induced Pluripotent Stem Cells”, by Kosuke Yusa, et al., and was published as a Nature Letter on October 20th, 2011.1 This is a proof-of-principle study for a new technology developed by the authors for eventual application in cell replacement therapy. The authors used a novel combination of zinc finger nuclease and piggy-Bac methodology in human induced-pluripotent stem cells (iPSCs) to correct a single point mutation in the α1-antitrypsin gene that is known to be responsible for α1-antitrypsin deficiency in humans. After successfully correcting the point mutation in several patient iPSC lines, the authors were able to differentiate the lines into fully hepatocyte-like cells in both structure and function. After in vivo transplantation into mouse livers, the hepatocyte-like cells distributed throughout the lobes of the liver and appeared to be functioning normally. The authors assert that their work is the first proof of principle for combining a genetic correction and human iPSCs in a way that is clinically applicable for cell therapies in which a patient’s own cells are isolated, subjected to corrective gene therapy, and then returned to the patient.
I selected this article for the midterm assignment because I was interested in learning more about research with human induced pluripotent stem cells and cell replacement therapy after the class session and paper discussion with Dr. Melissa Wong about intestinal stem cells. In addition to introducing a new method aimed at iPSC replacement therapy, the authors also test their method against several potential problems that could prevent it from being clinically applicable. Overall,...

... middle of paper ...


1. Yusa, K. et al. Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells. Nature 478, 391–394 (2011).
2. Fairchild, P. J. The challenge of immunogenicity in the quest for induced pluripotency. Nat. Rev. Immunol. 10, 868–875 (2010).
3. Lu, X. & Zhao, T. Clinical Therapy Using iPSCs: Hopes and Challenges. Genomics Proteomics Bioinformatics 11, 294–298 (2013).
4. Kim, A. & Pyykko, I. Size matters: versatile use of PiggyBac transposons as a genetic manipulation tool. Mol. Cell. Biochem. 354, 301–309 (2011).
5. Woltjen, K. et al. piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells. Nature 458, 766–770 (2009).
6. Urnov, F. D., Rebar, E. J., Holmes, M. C., Zhang, H. S. & Gregory, P. D. Genome editing with engineered zinc finger nucleases. Nat. Rev. Genet. 11, 636–646 (2010).

Need Writing Help?

Get feedback on grammar, clarity, concision and logic instantly.

Check your paper »

Stem Cell Technology: Hypoxia and Its Contributions to Regenerative Medicine

- Introduction Stem cell technology is developing rapidly to bring tissue and organ regeneration from the foreground of current research to the hands of physicians for therapeutic interventions of injuries. Though this field is rapidly progressing, several limiting factors have reduced the efficacy and survival of many transplanted cells. To understand the limitations, a deeper understanding of the chemo–mechanical environment of an injury is needed. Tissue and organ development from specific progenitor cells is tightly controlled by the surrounding biochemical environment....   [tags: science, medicine, stem cell research]

Research Papers
2526 words (7.2 pages)

A Research Study On Gene Therapy Essay

- The book talks about how upcoming trends in gene and cell therapy is meant for those who look for the valuable thread that runs through the arena of gene therapy, cell therapy, and tissue engineering, yet found other sources too specialized to undertake it. This book focuses on arming basic clinicians and scientists with this valuable thread so that they are better situated to tackle the weakening diseases currently plaguing mankind. The authors argue that gene and cell therapies are promising policies for managing acquired and genetic illnesses....   [tags: DNA, Gene, Human Genome Project, Genetics]

Research Papers
1089 words (3.1 pages)

Taking a Closer Look at Gene Therapy Essay

- Gene Therapy Genetic disorders have been plaguing people for ages and causing death. As of today in the year 2013 new information and research, and something called gene therapy, hope now exists for these less fortunate individuals. Gene therapy is a technique for correcting defective genes responsible for disease development. Research Scientist realizes it has been around for a while now and is getting more advanced with time. Experiments which is a ongoing process pertaining to gene therapy. Ethical issues are something that has been accompanying the procedure since it has been used....   [tags: genetic disorders, ethical issues]

Research Papers
942 words (2.7 pages)

Study of Bioinformatics, Proteomics, and Genomics Essay

- Executive Summary The current study illustrates about the important roles and the applications of bioinformatics, proteomics and genomics in biotechnology. The study clearly describes that bioinformatics, proteomics and genomics play significant roles in drug discovery and designing, agriculture, developing personalized medicines, biomarkers and in many other areas which will be highly useful in the present and also for the future generations of living organisms. Finally, the study also highlights on some points of recent studies on bioinformatics, proteomics and genomics by other researchers that have been conducted and their future capabilities are also clearly mentioned....   [tags: technology, genes, medical]

Research Papers
1994 words (5.7 pages)

Genetically Modified Animals Essay

- Genetically modified animals are an important category of the genetically modified organisms (EFSA, 2012). From EFSA and Paul Arnold (2013), genetic engineering of an animal involves manipulating or modifying the genetic code of the targeted animal to alter their original characteristics and introduce certain desired traits to the animal by adding to, substituting with or removing from genes of interest in a way that does not occur naturally (EFSA, 2012; Arnold, 2013). The genes of interest can be derived from another animal of the same species, an animal of another species, or even an organism of another kingdom (Arnold, 2013)....   [tags: dna, organisms, genes]

Research Papers
1791 words (5.1 pages)

Differences in Cancer Stem Cell Properties in Human Colon Cancer Cell Lines HCT116 and HT29

- Summary: Background and objective. Tumor heterogeneity is shown to be related to clinical outcome in cancer patients. The concept of a small subset of cancer stem cells being responsible for tumor relapse and metastasis comes out as a promising strategy for targeted cancer therapy. However, cancer stem cells are not easy to identify and isolate. The aim of this study was to determine the putative colon cancer stem cell subsets in human colon cancer cell lines HCT116 and HT29, which differ in their aggressiveness and differentiation capacity....   [tags: tumor heterogeneity, coln cancer]

Research Papers
1838 words (5.3 pages)

Identification and Characterization of Somatic Mutations Associated with Progression of Acute Myeloid Leukemia Having FLT3ITD

- ... We validated approximately 10-12 mutations per patient. We validated total 137 mutations in 132 genes from 11 cases by Sanger sequencing. These 137 mutations include single nucleotide variation (SNVs), small insertions and deletions (Indels). 60 mutations were present only in Dx and 20 were validated as Rel specific. In addition to this we observed 52 were present in both Dx and Rel. We identified and verified mutations in FAT1 in FLT3ITD subtype and studied the biology of these mutations in AML cells having FAT1 knockdown and overexpression resulting in a tumor suppressor-like activity....   [tags: cloning, cell. biochemistry, sequencing]

Free Essays
683 words (2 pages)

Genetic Screening Essay

- Genetic Screening Five year old Jacob Turner is a healthy boy without many cares in this world. His father takes sole care of him because his mother died suddenly. Genetic testing after death, showed a genetic mutation in Jacob's mothers genes that caused her to have an irregular heart. Unfortunately, Jacob has also inherited this mutation, but fortunately, this disorder can be controlled by medications. Now, Jacob's father has another problem. No insurance company will cover young Jacob because of his known heart irregularity....   [tags: DNA Science Genes Essays]

Free Essays
1967 words (5.6 pages)

The Science Of Stem Cell Therapy Essay

- By line The science around stem cells is an area that has great potential for treating human injury and diseases. News from media suggests stem cell treatment is close to being available to patients to treat wide varieties of diseases and injuries. However, further research is necessary before stem-cell-based therapy can be carried out in clinics safely and effectively. The Majority of stem cell treatments are in early stages of research and development, but with the recent rapid advancement in science and technology, stem cell therapies are likely to be just around the corner....   [tags: Stem cell, Cellular differentiation, Stem cells]

Research Papers
1536 words (4.4 pages)

The Genes Of A Cell Essay

- When studying the process of meiosis, one must understand the action of crossing over in the genes of a cell. This recombination helps to provide genetic diversity among the many different species of eukaryotes. The PRDM9 protein regulates this crossing over as well as the hotspots which exist along the genome. A “recombination hotspot” is an area along the gene where crossovers are more likely to occur. Cattle, the main focus of this study, have the PRDM9 protein which can be used to help identify and gain information regarding recombination in mammalian cells....   [tags: DNA, Gene, Genetic recombination, Genetics]

Research Papers
1354 words (3.9 pages)