User:TheEditor0702/CRISPR gene editing

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Discovery

2005 - Discovery of Cas9 and PAM: Alexander Bolotin at the French National Institute for Agricultural Research (INRA) discovered a CRISPR locus hat contained novel cas genes, significantly one that encoded a large protein known as CAS9.

2006 - Hypothetical Scheme of Adaptive Immunity: Eugene Koonin at the US National Center for Biotechnology information, NIH, proposed an explanation as to how CRISPR cascades as a bacterial immune system.

2007 - Experimental Demonstration of Adaptive Immunity: Philippe Horvath a Danisco France SAS displayed experimentally how CRISPR systems are an adaptive immune system, and integrate new phage DNA into the CRISPR array, which is how they fight off the next wave of attacking phage.

2012 - Development of CRISPR as a formal gene-editing tool: The research team led by the University of California, Berkeley, professor Jennifer Doudna and Umea University professor Emmanuelle Charpentier, were the first people to identify, disclose, and file a patent application for the CRISPR-Cas9 system needed to edit DNA. They also published their finding that CRISPR-Cas9 could be programmed with RNA to edit genomic DNA, now considered one of the most significant discoveries in the history of biology.

Recent Events

2023:

January - New Cas orthologs, engineered variants, and novel genome editing systems all emerged in 2022 and carried to the start of 2023. This in particular has revolutionized cardiovascular research, with specific advances in precise genome editing, such as base editing and prime editing.

March - CRISPR technology is being used in several clinical trials. A patient was enrolled in a clinical trial for Vertex's new exa-cel treatment which uses CRISPR to treat sickle cell disease.

Diabetes

On November 17, 2021 CRISPR therapeutics and ViaCyte announced that the Canadian medical agency had approved their request for a clinical trial for VCTX210, a CRISPR-edited stem cell therapy designed to treat type 1 diabetes. This was significant because it was the first ever gene-edited therapy for diabetes that approached clinics. The same companies also developed a novel treatment for type 1 diabetes to produce insulin via a small medical implant that uses millions of pancreatic cells derived from CRISPR gene-edited stem cells.

Neurological Diseases

CRISPR is unique to the development of solving neurological diseases for several reasons. For example, CRISPR allows researches to quickly generate animal and human cell models. This allows them to study how genes function in a nervous system. By introducing mutations that pertain to various diseases within these cells, researches can study the effects of the changes on nervous system development, function, and behavior. They can uncover the molecular mechanisms that contribute to these disorders, which is essential for developing effective treatments. This is particularly useful in modeling and treating complex neurological disorders such as Alzheimer's, Parkinson's, and epilepsy among other.

Alzheimer's Disease (AD) is a neurodegenerative disease categorized by neuron loss and an accumulation of intracellular neurofibrillary tangles and extracellular amyloid plaques in the brain. Three known pathogenic genes that cause early onset AD in humans has been identified, specifically amyloid precursor protein (APP), presenilin 1 (PSEN1), and presenilin 2 (PSEN2). Over 300 mutations have ben detected in these genes, resulting in an increase in total β-amyloid (Aβ), Aβ42/40 ratio, and/or Aβ polymerization.

Gyorgy et al. designed a specific gRNA for the APPswe mutation. The group that received a gRNA-treated mutation group had significantly lowered levels of Aβ40 compared to the group that didn't receive it. APPswe transgenic mice carry multiple copies of human APPswe mutation, so Gyorgy et al. delivered sgRNA and CAS9 into the primary cortical neurons from Tg2576 mice, which led to substantial decreases in Appswe. Thus, Gyorgy and associates proved that CRISPR-Cas9 can selectively break down the APP alleles, and thus decrease pathogenic Aβ.

In the case of Duchenne muscular dystrophy, the mutation responsible for the disease occurs in the dystrophin gene. CRISPR has been used to correct for this. Similarly, for Dravet syndrome, which is an epilepsy disorder, CRISPR has been used to correct the SCN1A gene mutation. Despite the progress that has been made, there are still challenges around using CRISPR. Due to the fact the brain is composed of a blood-brain barrier, it is difficult to transfer CRISPR components across this. However, recent advancements in nanoparticle delivery systems and viral vectors have shown promise in overcoming this hurdle. Looking in the future, the use of CRISPR in neuroscience is expected to increase as technology evolves.

Designer Babies

The advent of CRISPR-Cas9 gene editing technology has led to the possibility of creating "designer babies." While this technology has the possibility of creating eliminating certain genetic diseases, the earlier subsection discusses the issues between editing somatic cells and germline cells. Beyond this worry, significant ethical and societal concerns are raised due to access. Socioeconomic factors create a barrier to entry as to who can use the technology to "edit" their baby. Gene editing therapy isn't expected to be cheap, and neither is it for the any of the applications mentioned previously. While applications mentioned previously might have medical uses, specifically using gene editing for traits such as intelligence, physical appearance, and athletic ability would lead to a society where enhanced individuals have distinct advantages over those who aren't. This would further widen the gap between the rich and the poor.

The creation of designer babies could also lead to new form of racism and genetic discrimination. If parents can select which traits they want, they might opt out of certain ethnic features that are considered undesirable in society, and others that are. The presence of Eurocentric features dominating the universal beauty standard means these might be selected for. These would reinforce harmful stereotypes and lad to a new kind of racism, based on genetics that express "desirable features" that potentially pertain to a ingle race.

The ability to edit out undesirable traits can also contribute to ableism, a form of discrimination that favors able-bodied individuals. If traits such as physical disabilities can be edited out, this would lead to a society that devalues and stigmatizes those with disabilities. Treating those with such issues as something that needs to be fixed, potentially by CRISPR gene editing, ignores the diversity they bring to society on a physical, and intellectual level. Mental illnesses that are often labeled as a disability lead to individuals with unique traits that often produce prodigies within the arts.

The promise of eliminating genetic diseases via designer babies raises significant ethical and societal concerns. As we move forward with this technology, it is crucial we weigh the implications and prevent the exacerbation of inequality, racism, and ableism.