According to Cell Press, a website that publishes scientific journals about life, earth, and health sciences, gene editing is based on the use of engineered nucleases composed of specific DNA by erasing, replacings or inserting another DNA sequence to correct genetic disorders. According to Marylin Strathern, a british anthropologist who deals with issues of reproductive technologies, there has been an uprise of new technology used to help create genetically modified babies around the world. Many diseases such as cystic fibrosis, HIV, AIDS, and cancer are spreading around the world today. These technologies are going to create a happier and healthier society, free from genetic diseases. This creates the question how does the development of new technologies throughout the world help prevent diseases in genetically modified babies? Technologies such as the Zinc-fingers (ZFN), Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR), Transcription activator-like effector nucleases (TALEN), and genodicine have been designed to modify DNA sequences to help prevent unwanted diseases and disorders among unborn babies.
Zinc-fingers (ZFN) was the first known gene editing technology. Cell Press states that ZFN are DNA-binding proteins that reduce targeted DNA. ZFN have been used to genetically repair mutations associated with Parkinson’s disease within the Synuclein Alpha gene, which provides instructions for making a small protein called alpha-synuclein, in patient derived human cells. ZFN induced repair has also proven a potentially powerful strategy for fighting HIV and AIDs. ZFN have been used to confer HIV-1 resistance by disabling the HIV coreceptor and chemokine receptor type 5, a protein on the surface of white blood cells that is involved in the immune system that acts as a receptor for chemokines, in primary T cells and hematopoietic stem/progenitor cells, cells which are also apart of the immune system. ZFN is a very effective method but so is a newly developed technology called Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR).
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CRISPR has the power to modify the genes of human beings at infancy. Sikandar Hayat Khan, who works for a department of pathology states, CRISPR has two components, the SPR which is termed sometimes as spacers, which are hallmarked by varying and differing nucleotide sequences. Each one of the spacers represents a past exposure to foreign toxin. The CRI represents the genetic memory for bacteria and can be activated again once encountered to a similar foreign toxin. When these two concepts are put together it creates what we know today as the CRISPR (Khan). According to Brendan P. Foht, an editor of a website named The New Atlantis which improves public understanding of the social, political, ethical, and policy implications of modern science and technology, CRISPR is a technique that allows scientists to make precise edits to any DNA, whether bacterial or human. It allows researchers to simply change certain DNA sequences and modify gene function. Its applications include correcting genetic defects, treating and preventing the spread of diseases and improving crops. The enzymes that eliminate the targeted DNA are guided by short strands of RNA that can be customly designed for any set of chromosomes in a multicellular organism (Foht). Earlier genetic engineering methods require unalike enzymes to target different locations in the chromosomes, but by using RNA instead, CRISPR makes that targeting process much easier.
The most recently developed technology is called the Transcription Activator-like Effector Nucleases, known as TALEN, which restricts enzymes to be engineered to cut specific sequences of DNA. TALEN, like CRISPR, have high flexibility in design for specific targeted chromosomes and several examples of high editing efficiency. Other advantages are the design of TALEN are to target severe HIV DNA, which is relevant to latent infection, and encoding monomers, a molecule that can be bonded together with another molecule to form a molecular structure consisting entirely of a large number of similar units bonded together (Ronald Benjamin). Although TALEN isn't as effective as CRISPR, it is still improving everyday to help reach the goal of curing all unwanted genetic diseases in human beings.
According to Thomas Gaj, Charles A. Gersbach, and Carlos F. Barbas, professors in bioengineering at the University of Illinois, ZFN and TALEN are capable of correcting the underlying cause of diseases, therefore permanently eliminating the symptoms with precise genome modifications. ZFN induced homology directed repair (HDR), a mechanism in cells to repair double strand DNA lesions, has been used to directly correct the disease causing mutations associated with X- linked severe immune deficiency. ZFN, TALEN, and RNA-guided DNA endonucleases, an enzyme which leaves a chain by separating nucleotides, are transformative tools that have the potential to revolutionize biological research and affect personalized medicine. These new technologies have dramatically expanded the ability to manipulate and study model organisms, and correct the genetic causes behind many diseases. Through the years as technology continues to advance, people will discover ways to eliminate deadly diseases entirely, using gene editing, in an attempt to create a world with no sickness.
Genedicine is the first profit-oriented gene therapy medicine for the treatment of cancer that uses a shuttling system based to carry a p53 gene to limit cancer growth. There have been a complete one thousand and twenty approved gene therapy clinical trials within the world at the end of January 2005. Among these clinical trials, 66% were for the treatment of cancer. Of these cancer gene therapy trials, fifty eight used recombinant groups of DNA viruses encoding human tumor and cancer suppressor gene, p53. More than twenty kinds of cancer indications have been treated with p53, such as head and neck squamous cell carcinoma known to us as cancer in the lining of organs, lung cancer, breast cancer, liver cancer (Peng). There’s yet to be a cure for cancer. With the p53 gene, there is a chance that cancer can be reduced significantly and possibly even cured. According to Current Status of Gendicine in China: Recombinant Human Ad-p53 Agent for Treatment of Cancers In Clinical Trials and Applications, gendicine reduces the side effects caused by chemo and radiation therapy. Some important observations were that patients showed improved appetite and general health status approximately two days after receiving gendicine treatment. This is a positive clinical development for cancer patients who suffer from severe side effects caused by radio and chemotherapy (Peng). The mechanism leading to the improvement is not yet known; further study is underway to better understand the clinical implications.
Some solutions to stop diseases in the future generation are technologies such as the ZFN, CRISPR, TALEN, and genodicine. These technologies are known to be gene editing tools and therapy to help modify unwanted DNA sequences among humans. ZFN cures diseases such as HIV, AIDs and diseases involved in the immune system. CRISPR cures genetic defects such as disabilities. TALEN cures latent infection, hidden infections such as chickenpox and HIV. Genodince is a gene therapy that uses p53 to suppress tumor and cancer genes. CRISPR and genedicine are known to be the most effective solutions showing results in two days, saving lives and increasing life expectancy (Peng). There are still problems in all these technologies but as the years come, the effectiveness of them will increase greatly creating a happier and healthier society, free from genetic diseases.
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