In A 1st, Scientists Use Revolutionary Gene-Editing Tool To Edit Inside A Patient

In A 1st, Scientists Use Revolutionary Gene-Editing Tool To Edit Inside A Patient

Publication date: Mar 05, 2020

Scientists at the Casey Eye Institute, in Portland, Ore. , have have injected a harmless virus containing CRISPR gene-editing instructions inside the retinal cells of a patient with a rare form of genetic blindness.

KTSDesign/Science Photo Library/Getty Images hide caption toggle caption KTSDesign/Science Photo Library/Getty Images Scientists at the Casey Eye Institute, in Portland, Ore. , have have injected a harmless virus containing CRISPR gene-editing instructions inside the retinal cells of a patient with a rare form of genetic blindness.

KTSDesign/Science Photo Library/Getty Images For the first time, scientists have used the gene-editing technique CRISPR to try to edit a gene while the DNA is still inside a person’s body.

The groundbreaking procedure involved injecting the microscopic gene-editing tool into the eye of a patient blinded by a rare genetic disorder, in hopes of enabling the volunteer to see.

In those other cases, doctors removed cells from patients’ bodies, edited genes in the cells with CRISPR in the lab and then infused the modified cells back into the volunteers’ bodies to either attack their cancer or produce a protein their bodies are missing.

In this new experiment, doctors at the Casey Eye Institute in Portland, Ore. , injected (into the eye of a patient who is nearly blind from a condition called Leber congenital amaurosis) microscopic droplets carrying a harmless virus that had been engineered to deliver the instructions to manufacture the CRISPR gene-editing machinery.

The goal is that once the virus carrying the CRISPR instructions has been infused into the eye, the gene-editing tool will slice out the genetic defect that caused the blindness.

That would, the researchers hope, restore production of a crucial protein and prevent the death of cells in the retina, as well as revive other cells – enabling patients to regain at least some vision.

“It’s the first time the CRISPR gene-editing is used directly in a patient,” Pierce says.

Pierce, Albright and others stressed that only one patient has been treated so far and that the study, still at a very early stage, is designed primarily to determine whether injecting the gene-editing tool directly into the eye is safe.

“If we can do this safely, that opens the possibility to treat many other diseases where it’s not possible to remove the cells from the body and do the treatment outside,” Pierce says.

Concepts Keywords
Allergan Genetic code
Anemia CEP290
Beta Thalassemia Editas Medicine
Blindness CRISPR
Blood Leber’s congenital amaurosis
Body Treatment Gene therapy
Brain Genetic engineering
Cambridge Emerging technologies
Cancer Biotechnology
CRISPR Molecular biology
Dementia Life sciences
Dublin Branches of biology
Fluid Incisions
Francis Collins Blindness
Gene Congenital amaurosis
Genetic Retinal diseases
Genetic Code Class therapies diseases
Genetic Condition Lots diseases
Genetic Defect Rare genetic disorder
Genetic Disorder Gene editing machinery
Getty Images
Harvard
High Hopes
Huntington
Leber Congenital Amaurosis
Legally Blind
Massachusetts
Medicine
Muscle Diseases
Muscular Dystrophy
Mutation
Myotonic Dystrophy
NPR
Ophthalmology
Pennsylvania
Portland
Retina
Retinal
Retinal Diseases
Sickle
Spectrum
Surgeon
Virus

Semantics

Type Source Name
disease MESH retinal diseases
disease MESH myotonic dystrophy
disease MESH muscular dystrophy
disease MESH dementia
disease MESH brain disorders
disease MESH visual
disease MESH death
disease MESH Leber congenital amaurosis
disease MESH blindness
disease MESH genetic disorder
drug DRUGBANK Etodolac
disease MESH cancer
disease MESH sickle cell anemia
disease MESH beta-thalassemia

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