{"id":75359,"date":"2022-08-21T20:48:00","date_gmt":"2022-08-22T00:48:00","guid":{"rendered":"https:\/\/www.shortform.com\/blog\/?p=75359"},"modified":"2022-08-23T14:23:50","modified_gmt":"2022-08-23T18:23:50","slug":"the-future-of-gene-therapy","status":"publish","type":"post","link":"https:\/\/www.shortform.com\/blog\/the-future-of-gene-therapy\/","title":{"rendered":"Siddhartha Mukherjee: The Future of Gene Therapy"},"content":{"rendered":"\n

Can gene therapy cure any disease? How far are we from a future where gene therapy is mainstream? <\/p>\n\n\n\n

Scientists have already begun to explore the possibilities of gene manipulation with new technologies like stem cell research. However, progress is slow due to ethical and legal concerns, especially when it comes to modifying human genes. <\/p>\n\n\n\n

Keep reading to learn about the future of gene therapy. <\/p>\n\n\n\n\n\n\n\n

Gene Therapy Could Be the Future of Medicine<\/strong><\/h2>\n\n\n\n

Gene therapy\u2014using genetic engineering<\/a> to fix damaged or disease-causing genes\u2014offers promising treatments for diseases ranging from hemophilia and cystic fibrosis to cancer.<\/p>\n\n\n\n

(Shortform note: Safe and effective gene therapy is still a work in progress<\/a>. In the US, it\u2019s currently only available to patients who agree to participate in clinical trials.)<\/p>\n\n\n\n

One major area of study is in pluripotent stem cells<\/em>: immature cells that can be genetically manipulated to grow into any type of adult cell. While there are obvious ethical issues with harvesting immature cells from human embryos, doctors now believe it\u2019s possible to manipulate the genomes of adult cells so that they revert to stem cells. From there, the cells can grow into whatever\u2019s needed. In other words, doctors may be able to treat patients using stem cells harvested from their own bodies. <\/p>\n\n\n\n

Theoretically, doctors could use these stem cells to regenerate damaged nerves and organs, helping people to heal from injuries and diseases that are otherwise untreatable.<\/strong><\/p>\n\n\n\n

(Shortform note: Aside from exciting new treatment possibilities, stem cells are also useful to model how diseases progress and to test new drugs<\/a>. For example, a researcher could take a cell sample from a patient, grow that sample into new tissue, and observe how the disease affects it. The researcher could also use that sample to test experimental treatments without putting the patient\u2019s health at risk.)  <\/p>\n\n\n\n

Mukherjee also discusses CRISPRs: \u201cClustered Regularly Interspaced Short Palindromic Repeats\u201d\u2014in simple terms, repetitive and easily identifiable sequences of nucleotides. <\/p>\n\n\n\n

A gene editing technique called CRISPR-Cas9 targets those sequences using an enzyme called the Cas9 nuclease, allowing scientists to make precise cuts to DNA. That, in turn, allows specific sequences of DNA to be removed and other sequences to be inserted. In short, scientists can use this technique to make precise, controlled edits to a cell\u2019s DNA, thereby changing the genetic instructions encoded in it. <\/strong>Editing genes this way could potentially cure a wide range of genetic diseases, correct harmful mutations, and possibly even treat cancer. <\/p>\n\n\n\n

CRISPR<\/a><\/strong>: The Future of Gene Therapy<\/strong>?

While CRISPR-Cas9 is a powerful gene editing technique, it has several serious limitations<\/a> that scientists are still working to overcome:

Hard to scale. <\/strong>It\u2019s difficult to edit DNA in large numbers of cells at once, which severely limits CRISPR\u2019s usefulness as a treatment for widespread or systemic issues throughout the body. Not 100% effective. <\/strong>Even genes that are targeted by CRISPR may not show edited genomes as intended.Not 100% precise. <\/strong>Though rare, CRISPR can affect genes other than the intended targets. These unintended edits could, in theory, cause the sorts of harmful mutations that CRISPR-Cas9 is intended to correct\u2014for example, an uncontrolled mutation could cause cancer. <\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n

Germline Editing: Inheritable Changes<\/strong><\/h3>\n\n\n\n

Mukherjee says that gene therapy currently only affects the person it’s performed on and doesn\u2019t get passed to that person\u2019s children. However, it\u2019s theoretically possible to create a human embryo using genetically modified stem cells,<\/strong> if those stem cells can be converted into gametes (sperm and eggs). While that should be possible\u2014stem cells should be able to turn into any <\/em>type of cell\u2014the technique is still unproven. <\/p>\n\n\n\n

But if scientists could create genetically modified embryos in this way, it would mean all of that person\u2019s cells, including his or her gametes, would carry the modifications. Therefore, those changes would be passed down to any children the person had. <\/strong>At that point, Mukherjee says, we would have gone from editing a person\u2019s genes <\/em>to editing a person\u2019s genome<\/em>; in doing so, we\u2019d have created an entirely new type of organism, and potentially changed the gene pool forever. <\/p>\n\n\n\n