Mutated DNA Fixed in Patients: A Huge Step for Gene Therapy

A recent article by Gina Kolata, published on March 10, 2025 in The New York Times, discusses an exciting breakthrough in gene therapy. Scientists have succesfully fixed a genetic mutation with a single infusion carrying a treatment that precisely targets the mutated gene. This is for the first time that a mutated gene has been returned to normal. This study led by Beam Therapeauticals, focused on alpha-1 antitrypsin deficiency (AATD), which is a genetic disorder that causes serious lung and liver damage and instead of using traditional gene therapy which involve adding or silencing genes, scientists used a special version of CRISPR to edit a single DNA letter, like "correcting a typo" in the genetic code. The patients who received highest doses started making normal levels of the missing protein, which could mean stopping the disease before it gets worse.

        This coud be a turning point in gene therapy, proving fixing faulty DNA inside the body is possible and what's more interesting is that, according to Beam's CEO, John Evans there were no serious side effects. While researchers still have to study long term effects, if further trials confirms it's safety, this could save countless lives and maybe it could pave the way for treating other genetic disorders like sickle cell anemia. The idea of editing DNA, like fixing a typo is something that holds the potential to save many people suffering from genetic disorders.

Guided by CRISPR, prenatal gene editing shows proof-of-concept in treating disease before birth

In an article from Science Daily, scientists conducted gene editing to prevent a lethal liver disease in laboratory animals and offers to treat human congenital disease before birth. Researchers from Children's Hospital of Philadelphia used low-toxic DNA base editing tools to turn off the effects of a disease causing genetic mutation. In this study, scientists performed prenatal gene editing to improve liver function and to prevent neonatal death in a group of mice that had been engineered with a mutation causing the lethal disease called hereditary tyrosinemia type 1 (HT1). In humans, HT1 usually occurs during infancy and treated with a medicine called nitisinone and strict diet. But, when the treatment fails the patients are in severe risk of liver failure or cancer. Scientists suggested that prenatal gene editing can be used to prevent disease like HT1 and many other congenital diseases. The research used base editor 3 (BE3) and a modified CRISPR associated with protein 9 (CRISPR-Cas 9) tool to carry an enzyme to a particular genetic location in the liver cells of the fetal mice. The enzyme modified the targeted genetic sequence of liver cells chemically by changing the type of DNA bases into another. As a result, the mice showed reduced level of cholesterol and had improved liver function.

A future application for DNA base editing could be correcting disease-causing mutations and to improve functions of organs beyond liver. I think this technique would be very useful for doctors to treat diseases during early pregnancy to ensure the health of the fetus.

DNA Editing, Are Humans Ready?

New technology has arrived in which scientist utilized CRISPR/Cas as a way to control the genetic makeup of DNA. CRISPR is mainly used in RNA as a sort of gene editing software while Cas is a bacteria derived from protein. The ability to change the DNA coding of a person could be the answer to life's greatest struggles such as diseases such as Aids or even a cure for cancer as well. Some scientist speculates that with this new revelation that it could be used to create what is called a "designer baby", in which the genetic code of an unborn child could be modified so that the parent can have a child to exactly their specifications.   

Though many human diseases are caused by DNA mutation treatment for the side effects rarely focus on the genetic makeup for results. This idea of genetic modification seems well beyond our time and is already causing controversy. Such that the study itself has been called on to halt all further research in fear of science being beyond the grasp of human society.