Genterapi mod arvelige øjensygdomme

Line Kessel; Mette Bertelsen; Kristian Klemp; Ulrik Correll Christensen

Genterapi mod arvelige øjensygdomme

Line Kessel, Mette Bertelsen, Kristian Klemp, Ulrik Correll Christensen

Abstrakt

Inherited retinal disorders (IRD) are a common cause of severe visual impairment among children and young adults in Denmark. Gene therapy with voretigene neparvovec for a specific, and in Denmark common, cause of IRD (RPE65-related retinal dystrophy) was implemented as standard clinical practice in 2020 as the first of its kind. Twelve Danish patients have been treated with very positive outcomes. Genetically based therapies for other genetic causes of IRD are underway in clinical trials and are expected to change the outlooks for patients who would otherwise become blind early in life.

Bidragets oversatte titel	Gene therapy for hereditary eye diseases
Originalsprog	Dansk
Artikelnummer	V07220453
Tidsskrift	Ugeskrift for Laeger
Vol/bind	184
Udgave nummer	45
ISSN	0041-5782
Status	Udgivet - 7 nov. 2022

Emneord

Child
Young Adult
Humans
Genetic Therapy
Retinal Dystrophies/genetics
Eye Diseases, Hereditary/genetics
Mutation

Adgang til dokumentet

https://ugeskriftet.dk/videnskab/genterapi-mod-arvelige-ojensygdomme

Citationsformater

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title = "Genterapi mod arvelige {\o}jensygdomme",

abstract = "Inherited retinal disorders (IRD) are a common cause of severe visual impairment among children and young adults in Denmark. Gene therapy with voretigene neparvovec for a specific, and in Denmark common, cause of IRD (RPE65-related retinal dystrophy) was implemented as standard clinical practice in 2020 as the first of its kind. Twelve Danish patients have been treated with very positive outcomes. Genetically based therapies for other genetic causes of IRD are underway in clinical trials and are expected to change the outlooks for patients who would otherwise become blind early in life.",

keywords = "Child, Young Adult, Humans, Genetic Therapy, Retinal Dystrophies/genetics, Eye Diseases, Hereditary/genetics, Mutation",

author = "Line Kessel and Mette Bertelsen and Kristian Klemp and Christensen, {Ulrik Correll}",

year = "2022",

month = nov,

day = "7",

language = "Dansk",

volume = "184",

journal = "Ugeskrift for Laeger",

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TY - JOUR

T1 - Genterapi mod arvelige øjensygdomme

AU - Kessel, Line

AU - Bertelsen, Mette

AU - Klemp, Kristian

AU - Christensen, Ulrik Correll

PY - 2022/11/7

Y1 - 2022/11/7

N2 - Inherited retinal disorders (IRD) are a common cause of severe visual impairment among children and young adults in Denmark. Gene therapy with voretigene neparvovec for a specific, and in Denmark common, cause of IRD (RPE65-related retinal dystrophy) was implemented as standard clinical practice in 2020 as the first of its kind. Twelve Danish patients have been treated with very positive outcomes. Genetically based therapies for other genetic causes of IRD are underway in clinical trials and are expected to change the outlooks for patients who would otherwise become blind early in life.

AB - Inherited retinal disorders (IRD) are a common cause of severe visual impairment among children and young adults in Denmark. Gene therapy with voretigene neparvovec for a specific, and in Denmark common, cause of IRD (RPE65-related retinal dystrophy) was implemented as standard clinical practice in 2020 as the first of its kind. Twelve Danish patients have been treated with very positive outcomes. Genetically based therapies for other genetic causes of IRD are underway in clinical trials and are expected to change the outlooks for patients who would otherwise become blind early in life.

KW - Child

KW - Young Adult

KW - Humans

KW - Genetic Therapy

KW - Retinal Dystrophies/genetics

KW - Eye Diseases, Hereditary/genetics

KW - Mutation

M3 - Review

C2 - 36345897

VL - 184

JO - Ugeskrift for Laeger

JF - Ugeskrift for Laeger

SN - 0041-5782

IS - 45

M1 - V07220453

ER -

Genterapi mod arvelige øjensygdomme

Abstrakt

Emneord

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Citationsformater