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Vol 25, No. 03, March 2021   |   Issue PDF view/purchase
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Rising Prevalence of Genetic Disorders among Global Population & Subsequent Interest in Gene Editing Technologies
As a technology, gene editing has demonstrated myriad benefits for the medical industry over the years. Gene editing has tremendous potential in the treatment of inherited diseases, generation of more resilient crops, and even the detection of varying species in the environment.
by Saloni Walimbe

Genetics have widespread implications on human health over the life course. Gene anomalies can therefore result in various genetic disorders that can occur at any given point in a person’s life. Studies suggest that nearly 5000-8000 rare disorders have been recorded worldwide, affecting approximately 6 to 8 percent of the world population. Furthermore, it is estimated that around 40 percent of these rare diseases may be caused due to genetic factors.

Genetic disorders can arise due to a number of abnormalities such as additional chromosomes or gene mutations. Primitive medical treatments were ill-equipped to determine the actual impact of these abnormalities on a patient’s DNA, making the diseases unpredictable.

However, modern medicine has emerged as a solution to these issues, by giving rise to a slew of novel methods designed to identify the potential outcomes of genetic disorders. These methods are a result of unwavering medical research efforts from practicing physicians, researchers, and advanced-degreed nurse practitioners alike.

Through the collection and thorough analysis of evidence-based statistical data, individuals and authorities in the medical field have made significant strides in the development of targeted gene therapies, aimed at identifying, treating, and possibly even preventing the onset of certain genetic diseases. Gene editing, in particular, is emerging as one of the foremost features of gene therapies in recent years.

Gene Editing – an overview

Gene editing, also known as genome editing, is a method that enables scientists to modify the DNA of various organisms, including animals, bacteria, and plants. The method includes the use of several different technologies, which act as scissors, designed to cut DNA at specific spots. Once this is achieved, scientists are able to add, remove or replace DNA at the place where it was cut, thus altering the genetic makeup of the organism in such a way that it can either treat a pre-existing genetic condition or prevent the onset of the disease altogether.

The gene editing market has undergone significant evolutions throughout its history, each of which highlights the remarkable progress achieved in the field, as well as the crucial role of basic science research in the creation of effective research tools and disease treatments.

The earliest known gene editing technologies dates back to the 1900s, with the emergence of a method known as homologous recombination. Homologous recombination refers to the exchange of genetic data between two similar, or “homologous” DNA strands. The development of this genome editing technique truly commenced in the late 1970s, following scientists’ observations regarding the ability of organisms such as yeast to naturally carry out homologous recombination.

The 1990s witnessed the rise of ZFN (zinc-finger nucleases), which scientists began to use, in order to enhance the specificity of gene editing and to mitigate off-target edits. ZFN structures are engineered using naturally occurring proteins prevalent mainly in eukaryotic organisms. These proteins were engineered by scientists to bind with specific DNA sequences within the genome and then cut the DNA.

In the modern era, however, gene editing is considered to be nearly synonymous with the editing tool CRISPR (Clustered regularly interspaced short palindromic repeats), which has made the genome editing process easier than ever. One of the main factors that set CRISPR apart from its predecessors in gene editing history, is its simplicity.

Although DNA sequences associated with CRISPR were observed in bacteria as far back as the early 1990s, it wasn’t until the 2000s that the true potential of the technology to recognize and cut specific genome sequences came to light. The CRISPR technology uses the Cas9 protein, a protein with strong DNA cutting abilities, to deliver a gene editing method that is much faster, simpler, economical, and more precise than any other method. CRISPR is also observed to occur in nature, where it is used by bacteria as the immune system to eliminate invading viruses. However, over the years it is gradually being adopted for use in laboratories as well.

As a technology, gene editing has demonstrated myriad benefits for the medical industry over the years, including strong potential in the treatment of inherited diseases, a better understanding of specific gene outcomes, generation of more resilient crops, and even the detection of varying species in the environment.

Emerging trends and developments driving progress in the gene editing industry

In the modern era, advancements and persistent efforts by key players have given rise to myriad technologies in the gene editing business landscape, built on the prevailing CRISPR/Cas9 system, with a steadily broadening application scope.

To illustrate, in June 2020, researchers at Johns Hopkins Medicine announced the successful use of light as the trigger for precise cuts in genomic material, in a series of experiments conducted on human cancer cell lines. The novel technology involved the use of a molecular scalpel called CRISPR and observed the repairing effect of specialized cell proteins on the exact spots where the gene was cut. The results of the experiments were able to not only unearth new details pertaining to the DNA repair process but are also likely to expedite and assist in a better understanding of DNA activity related to aging and several cancers.

Gene editing technologies have also shown immense promise in the current COVID-19 era, where scientists worldwide are involved in a race against time to develop an effective vaccine against the deadly virus.

For instance, scientists at Stanford University have created an antiviral agent to combat COVID-19, by targeting and eliminating specific genetic strands of the virus within a human cell. The novel tool, dubbed Prophylactic Antiviral CRISPR in human cells or PAC-MAN, was initially developed as a treatment for influenza. The PAC-MAN technology comprises a strand of guide RNA as well as an enzyme and has demonstrated significant potential in laboratory tests involving SARS-CoV-2-infected human lung cells.

Similarly, a team of researchers, spearheaded by Nobel Prize winner Jennifer Doudna, has made significant strides in COVID-19 testing, by developing a unique test that can detect infection in just 5 minutes. The test, which involves the use of a gene-editing technology and a modified mobile phone camera with a portable device fitted with collection optics and low-cost laser illumination, is aimed at eliminating the need for expensive lab equipment for COVID-19 testing and offering patients a chance to administer the test even within their homes.

Contributions by leading industry vendors as well as research institutes towards gene editing technologies

Gene therapies are entering a brand-new era across the globe, as a result of consistent contributions from major gene editing market players as well as research organizations worldwide. Following a string of successful clinical trials, the U.S government decided, in 2018, to lift the previously stringent NIH special oversight rules on gene therapy research, and began to take various gene therapy drugs into consideration for approval in the same way as other medical developments. In fact, in January 2019, the US FDA (Food and Drug Administration) had taken over 800 applications into consideration, for the preclinical development or clinical trials for gene therapies.

Massive support for these efforts has been observed mainly by leading Japanese start-ups in the gene therapy and gene editing industries. These businesses began to gain prominence following 2014 and have started to attract millions of dollars’ worth of investor and government funding in recent years. Notable among these are two 2015 entrants in the market, EditForice, and EdiGene, as well as 2017 entrant Bio Palette, which is rapidly making its way to the global stage, via a cross-licensing deal with Beam Therapeutics, a start-up leveraging Harvard University and MIT-based technologies, in 2019, aimed at developing mutagenesis-based gene editing technologies.

Similar efforts have been observed in Taiwan as well, where the region’s Ministry of Science and Technology (MOST) has initiated a collaboration with a number of global institutions such as TVGH (Taipei Veterans General Hospital), UCLA (University of California, Los Angeles), and NYMU (National Yang-Ming University). The team has set a collective objective to combine SMNPs (self-assembled supramolecular nanoparticles) and CRISPR/Cas9 technology, in order to find a universal and secure treatment plan for inherited diseases such as Beta-Thalassemia and Sickle cell disease, characterized by decreased production or structural changes of β-globulin. Results from the study revealed the integration of a functional HBB gene into human blood cells, via mediation by SMNPs and CRISPR/Cas9, using Ad-SiNWS (Ad-grafted silicon nanowire substrates), thereby facilitating the successful delivery of the RNP-SMNPs complex into an in-vitro model, a significant milestone for the research effort.

Technological breakthroughs characterizing a promising future outlook for genome editing

Various successful preclinical developments, as well as the rapid progression of gene editing in clinical trials, have created a significant sense of optimism for the gene editing industry regarding the future of the field.

Persistent developmental effort and advancements in the industry are likely to open up lucrative avenues for novel technologies that will broaden the scope of genome editing over the years. For instance, the rise of alternative gene editing technologies, like targetable site-specific recombinases that do not rely on DSB (double-strand breaks) creation, advanced CRISPR technologies with unique characteristics, and DNA-guided nuclease systems are set to make significant headway in advancing genetic therapies in the years ahead.

Epigenomic editing, which involved DNA-targeting platforms for specific modification of chromatin structure or gene regulation, is also generating commendable growth trajectories for manipulation of genomes cell and gene therapies and is thus a key contributor to ensuring the precision and safety of the next generation of genome editing systems.

In essence, gene editing has redefined cell and gene therapy and has played a key role in the renewed interest in the field over recent years. While there are still some translational and fundamental parameters to be examined for realizing the full potential of these technologies, gene editing is nevertheless paving the way for a brighter future for gene therapies in the years to come.

References:

  • Akriti Anand, India Today, Detect coronavirus in just 5 min: Nobel Prize winner develops unique Covid test. (October 9, 2020). Retrieved from: https://www.indiatoday.in/science/story/now-coronavirus-infection-detected-just-5-minutes-study-1729998-2020-10-09
  • Biospectrum, Taiwan accelerates pace of gene-editing technologies into clinical settings. (November 6, 2020). Retrieved from: https://www.biospectrumasia.com/news/98/17074/taiwan-accelerates-pace-of-gene-editing-technologies-into-clinical-settings.html
  • Sumant Ugalmugle and Rupali Swain, Global Market Insights, Gene Editing Market Size and Share (Global Report 2026). (September 2020). Retrieved from: https://www.gminsights.com/industry-analysis/gene-editing-market

About the Author

An avid reader since childhood, Saloni is currently following her passion for content creation by penning down insightful articles relating to global industry trends, business, and trade & finance. With an MBA-Marketing qualification under her belt, she has spent two years as a content writer in the advertising field. Aside from her professional work, she is an ardent animal lover and enjoys movies, music and books in her spare time.

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