The coming of age of gene therapy: A review of the past and path forward

January 11, 2018, NIH/National Heart, Lung and Blood Institute
Media Availability: The coming of age of gene therapy: A review of the past and path forward
A new gene is injected into an adenovirus vector, which is used to introduce the modified DNA into a human cell. If the treatment is successful, the new gene will make a functional protein. Credit: US National Library of Medicine

After three decades of hopes tempered by setbacks, gene therapy—the process of treating a disease by modifying a person's DNA—is no longer the future of medicine, but is part of the present-day clinical treatment toolkit. The Jan. 12 issue of the journal Science provides an in-depth and timely review of the key developments that have led to several successful gene therapy treatments for patients with serious medical conditions.

Co-authored by Cynthia E. Dunbar, M.D., senior investigator at the Hematology Branch of the National Heart, Lung and Blood Institute (NHLBI), part of the National Institutes of Health, the article also discusses emerging genome editing technologies. According to Dunbar and her colleagues, these methods, including the CRISPR/Cas9 approach, would provide ways to correct or alter an individual's genome with precision, which should translate into broader and more effective gene therapy approaches.

Gene therapy is designed to introduce genetic material into cells to compensate for or correct abnormal genes. If a mutated gene causes damage to or spurs the disappearance of a necessary protein, for example, gene therapy may be able to introduce a normal copy of the gene to restore the function of that protein.

The authors focused on the approaches that have delivered the best outcomes in gene therapy so far: 1) direct in vivo administration of viral vectors, or the use of viruses to deliver the therapeutic into ; and 2) the transfer of genetically engineered blood or bone marrow stem cells from a patient, modified in a lab, then injected back into the same patient.

Originally envisioned as a treatment solely for inherited disorders, gene therapy is now being applied to acquired conditions such as cancer. For example, the engineering of lymphocytes, , that can be used in the targeted killing of .

In 2017, a steady stream of encouraging clinical results showed progress in for hemophilia, sickle-cell disease, blindness, several serious inherited neurodegenerative disorders, an array of other genetic diseases, and multiple cancers of the bone marrow and lymph nodes.

Three gene therapies have been approved by the U.S. Food and Drug Administration in the past year, and many more are under active clinical investigation. The authors looked to the future of gene therapies, and the challenges of delivering these complex treatments to patients.

Explore further: Will AAV vectors have a role in future novel gene therapy approaches?

More information: "Gene therapy comes of age" Science (2018). science.sciencemag.org/cgi/doi … 1126/science.aan4672

Related Stories

Will AAV vectors have a role in future novel gene therapy approaches?

March 20, 2017
Recombinant adeno-associated virus (rAAV) vectors for delivering therapeutic genes have demonstrated their safety in multiple diseases and clinical settings over the years and are a proven and effective tool that can be used ...

Gene editing of blood stem cells can correct disease-causing mutations

September 23, 2016
Recent advances in gene editing technology, which allows for targeted repair of disease-causing mutations, can be applied to hematopoietic stem cells with the potential to cure a variety of hereditary and congenital diseases. ...

Testing the efficacy of new gene therapies more efficiently

March 21, 2017
Using a new cellular model, innovative gene therapy approaches for the hereditary immunodeficiency Chronic Granulomatous Disease can be tested faster and cost-effectively in the lab for their efficacy. A team of researchers ...

Genetic treatment for blindness may soon be reality

November 11, 2017
Patients who had lost their sight to an inherited retinal disease could see well enough to navigate a maze after being treated with a new gene therapy, according to research presented today at AAO 2017, the 121st Annual Meeting ...

Gene therapy for liver disease advancing with the help of adeno-associated viral vectors

December 30, 2016
Liver-directed gene therapy delivered using adeno-associated viral (AAV) vectors to treat diseases such as hemophilia have advanced into human testing. The potential for continued technological improvements to expand the ...

What makes cancer gene therapy so groundbreaking?

July 19, 2017
On July 12, a Food and Drug Administration panel unanimously recommended approval for the first-ever gene therapy treatment for cancer. The treatment, known as CTL019, is a T-cell therapy developed by the pharmaceutical company ...

Recommended for you

Researchers identify new genetic disorder

September 21, 2018
Researchers from Michigan State University College of Human Medicine and physicians from Spectrum Health have identified for the first time in a human patient a genetic disorder only previously described in animal models.

Test could detect patients at risk from lethal fungal spores

September 20, 2018
Scientists at The University of Manchester have discovered a genetic mutation in humans linked to a 17-fold increase in the amount of dangerous fungal spores in the lungs.

Researchers identify a new cause of childhood mitochondrial disease

September 20, 2018
A rapid genetic test developed by Newcastle researchers has identified the first patients with inherited mutations in a new disease gene.

Why some human genes are more popular with researchers than others

September 18, 2018
Historical bias is a key reason why biomedical researchers continue to study the same 10 percent of all human genes while ignoring many genes known to play roles in disease, according to a study publishing September 18 in ...

Class of neurological disorders share 3-D genome folding pattern, study finds

September 18, 2018
In a class of roughly 30 neurological disorders that includes ALS, Huntington's Disease and Fragile X Syndrome, the relevant mutant gene features sections of repeating base pair sequences known as short tandem repeats, or ...

Researchers resolve decades-old mystery about the most commonly mutated gene in cancer

September 18, 2018
The most commonly mutated gene in cancer has tantalized scientists for decades about the message of its mutations. Although mutations can occur at more than 1,100 sites within the TP53 gene, they arise with greatest frequency ...

1 comment

Adjust slider to filter visible comments by rank

Display comments: newest first

gzurbay
not rated yet Jan 13, 2018
Sounds like science fiction, but is becoming the tip of the spear. As such there are things to consider. If simple gene replacement is the answer, will there be no fault? Will therapy be only for the well-to-do or a universal provided procedure. What of when the agent comes out of a vending machine and anyone can have disease eliminated? Will all CITIZENS get one free Crisprthery and have to pay for additional out of pocket? Will criminals in prison get a freebie??

If life span is enhanced radically how do we compensate for family structure - resources - retirement plans, jail sentences....

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.