Gene editing is an advanced technique for making small adjustments to DNA in living organisms. It permits researchers to disable target genes, repair harmful mutations and alter gene activity.
Gene editing has generated tremendous enthusiasm due to its potential to treat many common genetic diseases, such as cystic fibrosis, sickle cell anemia and muscular dystrophy.
Costs
Gene editing is no different, and like any new technology it comes with costs. These include research and development to create safe and effective therapies as well as regulatory testing to guarantee their safety.
Gene editing could have many potential negative consequences for society, such as inequality and unintended health risks that are still uncertain. About two-thirds of Americans express concern about this possibility while half are enthusiastic.
On the other hand, many people believe gene editing is no different from other ways humans strive for improvement. About three out of ten adults report positive outcomes such as increased confidence in recipients after receiving genetic modifications.
But there are concerns about the costs of gene editing. Some therapies, such as Novartis’ hemophilia A gene therapy Valrox, which has a price tag of $2 million per treatment, seem quite costly.
Accessibility
Editing genes is like having the “find and replace” feature on a computer – it allows scientists to correct errors and fix genetic mutations. It’s an invaluable new resource for scientists, plus it may even prevent children from developing certain diseases.
Though CRISPR technology has become widely used in labs around the world, there remain some obstacles to its widespread adoption, particularly regarding accessibility and scalability. For instance, performing multiple edits per experiment would be ideal for expediting biological discovery and product development but can be challenging to accomplish.
Despite these difficulties, there are various efforts to make CRISPR/Cas9 more accessible to researchers and manufacturers. One approach, called CRISPR-chrom, combines Cas9 orthologs with chromatin modulating peptides (CMPs) which improve local accessibility and editing efficiency at refractory sites. Another combination of Cas9-TV and dsgRNA increases Cas9 editing efficiency both open and closed chromatin regions equally. Furthermore, using catalytically dead SpCas9 at proximal sites can further boost Cas9 editing efficiency in vivo.
Safety
Scientists around the globe are exploring both the advantages and potential hazards of gene editing, from restoring genetic diversity in plants to curing hereditary diseases. While many experts believe that its advantages outweigh its drawbacks, some worry it could lead to “designer humans.”
CRISPR-based genome editing is highly dependent on specificity–that is, how precisely genes can be targeted and edited. To improve specificity, nucleases and repair templates need to be designed thoughtfully, off-target effects evaluated, and viral vectors utilized with care.
Recent research by Chiarle, Tao and colleagues demonstrates how CRISPR causes double-strand DNA breaks which may allow mobile elements to insert themselves in unexpected locations of the target sequence. They therefore recommend adding a check for retrotransposition into standard safety tests as an extra precaution.
Scientists should continue to explore and minimize the potential side effects of germline editing before it is used in human clinical trials. Furthermore, countries should place strict policy and regulatory restrictions on its use until such time as they understand and comprehend its risks.
Ethics
Genome editing is the process of altering DNA, the genetic code that makes up living organisms. It can be employed to disable target genes, correct harmful mutations or regulate gene activity.
Gene editing can also be employed to produce novel proteins or DNA sequences. Different technologies exist for this purpose, such as CRISPR-Cas9 and TALENs.
Though these techniques are highly precise, they may cause unintended effects. These consequences could be hazardous if they disrupt healthy genes or regulatory DNA.
Some researchers and bioethicists believe gene editing to be unethically acceptable. On the other hand, others maintain genome editing is necessary for curing diseases but should only be employed therapeutically.
The Nuffield Council on Bioethics has recommended that gene editing should only be morally permissible if it serves to secure and ensure the wellbeing of any potential human child. This principle would allow any specific application of gene editing to be legal as long as it promotes individual wellbeing and social solidarity.