Understanding Gene Editing Therapy

Understanding Gene Editing

What Makes Gene Editing Different

Gene editing is a type of gene therapy that works by making precise changes to the DNA of target cells. Different types of gene editing—such as CRISPR and TALENs—act like molecular scissors to cut and repair the genetic code. These tools allow scientists to fix the root cause of certain genetic diseases with incredible precision.

How Gene Editing is Changing Lives

A journey from pain to possibility with gene editing therapy.

For years, Victoria Gray lived with the constant pain and complications of sickle cell disease (SCD)—a genetic blood disorder that shaped every part of her life. In 2019, she became the first person in the U.S. to receive an experimental gene editing therapy using CRISPR technology.

Her edited stem cells have increased fetal hemoglobin, a healthy form of the protein her body needed.

“It meant a new beginning. It is more than I ever dreamed of for everything [the symptoms] to be gone.”

—Victoria Gray

Patient impact story image highlighting Victoria's life improvement for sickle cell disease via gene editing therapy

What It Takes to Make It Work

Turning gene editing into safe treatments requires precision, safeguards, and trust.

How Gene Editing Works

Gene editing tools precisely change faulty DNA inside living cells. CRISPR is the most widely known, but other types of gene editing include TALENs, zinc finger nucleases, and newer methods like base and prime editing. Base editing allows for a direct correction of some “letters” of DNA, without requiring a double-stranded break (DSB) of the DNA. PRIME editing is a newer method that can correct the entire “alphabet’ of DNA without a DSB. Each tool differs in how it cuts, repairs, or rewrites DNA.

Developers must ensure the therapy reaches the right cells, makes only the intended change, and remains safe over time.

On the Horizon for Gene Editing

Gene editing therapies are making incredible progress and hold the potential to cure diseases once thought untreatable. New techniques are expanding the possibilities of gene editing therapy, from base editing to epigenetic editing approaches in early trials. Each opens new opportunities while raising important considerations about safety and public trust.

To help address these issues, ARM facilitates collaboration across the CGT community, creating opportunities to align on standards and evidence on important topics like gene editing platforms.

Technologies Behind Gene Editing

Zinc Finger Nucleases (ZFN) – Binds to specific DNA sequences through three-base-pair sequences to inactivate mutated genes that cause disease.

Transcription Activator-Like Effector Nucleases (TALEN) – Engineered nucleases that can bind and cleave DNA by recognizing single nucleotides.

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) – Cuts parts of the DNA with a Cas9 enzyme to add a new sequence or inactivate genes.

Base Editing - Allows for precise modifications to individual DNA letters in a sequence.

Prime Editing - A technique that corrects the entire “alphabet’ of DNA without a double-stranded break.

Epigenetic Editing – A technology that aims to alter gene expression without altering the DNA through controlling epigenetic activity.

Overview of gene editing therapy methods such as epigenetic editing tools, prime editing, base editing, ZFN, CRISPR, and TALEN

Gene Editing Therapy