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samillingworth.com
👁️ Gene Therapy Restores Vision in Children

A first-in-human study shows that gene therapy improved vision in young children with severe AIPL1-related retinal dystrophy, preserving retinal structure and enhancing visual function.

🔗 www.thelancet.com/journals/lan...

#GeneTherapy #SciComm 🧪
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...
www.thelancet.com
February 22, 2025 at 7:53 PM
scinews.bsky.social
Genetic therapy gives infants life-changing improvements in sight. #TheLancet

"Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study"

www.thelancet.com/journals/lan...
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...
www.thelancet.com
February 21, 2025 at 5:27 AM
thelancetneuro.bsky.social
New in @thelancet.bsky.social, a first-in-human gene therapy trial in children with AIPL1-associated severe retinal dystrophy. www.thelancet.com/journals/lan...
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...
www.thelancet.com
February 26, 2025 at 9:10 AM
childvisionlab.bsky.social
Thrilled to see this monumental work in @thelancet.bsky.social! A single subretinal injection led to staggering improvements in nearly blind children, while behavioural and EEG data from our lab reveal a dramatic boost in function and cortex activity. A major leap for vision restoration! t.ly/fvdQy
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...
t.ly
February 21, 2025 at 11:25 AM
thelancet.com
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study

🔗 tinyurl.com/bdeezck3
Research in context panel for "Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study". Copyright: 2024 The Author(s). Published by Elsevier Ltd. CC BY 4.0.
February 24, 2025 at 2:05 PM
cerebrodigital.net
Un ensayo clínico en The Lancet logró restaurar visión parcial en niños con ceguera genética mediante terapia génica con el gen AIPL1. El avance abre nuevas posibilidades para tratar otras enfermedades oculares y condiciones hereditarias complejas.
October 3, 2025 at 9:30 AM
petetheteapot.bsky.social
Another success for gene therapy!

From The Lancet: Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study

www.thelancet.com/journals/lan...

#ophthalmology #genetherapy
@
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...
www.thelancet.com
February 21, 2025 at 10:17 AM
profsimonwad.bsky.social
A new paper hitting the press: Gene therapy for AIPL-1 retinal dystrophy. Almost all kids with this can only perceive light, at best. Getting early will help with vision also neurodevelopment and psychosocial aspects. 🧬 ⭐ 👁

www.thelancet.com/journals/lan...
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...
www.thelancet.com
April 14, 2025 at 7:06 AM
auditorynerves.bsky.social
Open access study here:

Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study. 🧪👁️🧬 #medsky
www.thelancet.com/journals/lan...
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...
www.thelancet.com
February 21, 2025 at 8:50 AM
correiocb.bsky.social
🧪 Seleção de DNA traz chances para crianças cegas

A terapia consiste em uma injeção de cópias saudáveis do gene AIPL1 na retina. Essas cópias são transportadas em um vírus inofensivo.
Seleção de DNA traz chances para crianças cegas
A terapia consiste em uma injeção de cópias saudáveis do gene AIPL1 na retina. Essas cópias são transportadas em um vírus inofensivo, o que permite que o material genético penetre nas células retinian...
www.correiobraziliense.com.br
February 21, 2025 at 12:20 PM
mariosgeorgakis.bsky.social
7⃣Gene therapy for AIPL1 retinal dystrophy restores vision

In 4 children (1-2.8 y) with AIPL1 severe retinal dystrophy, subretinal injection of rAAV vector carrying the human AIPL1 gene led from legal blindness to significant gains in visual acuity over 3.5 years

🔗 www.thelancet.com/journals/lan...
February 24, 2025 at 4:09 PM
seeingwithsound.bsky.social
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study www.thelancet.com/journals/lan...
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...
www.thelancet.com
February 21, 2025 at 3:19 PM
sciencex.bsky.social
Gene therapy using rAAV8.hRKp.AIPL1 shows promise in improving vision and preserving retinal structure in young children with AIPL1-associated retinal dystrophy, with sustained benefits observed. doi.org/g86b7x
Gene therapy can improve vision in young children with AIPL1-associated retinal dystrophy
Researchers from the NIHR Moorfields Biomedical Research Centre and University College London have found that gene therapy improved visual acuity and preserved retinal structure in young children with AIPL1-associated severe retinal dystrophy.
medicalxpress.com
February 25, 2025 at 9:02 PM
ianmccormick.me
New paper out today with some very cool results - Gene therapy in children with AIPL1-associated severe retinal dystrophy…improved visual acuity and functional vision and evidence of some protection against progressive retinal degeneration, without serious adverse effects. #EyeSky #OphthoSky
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...
www.thelancet.com
February 21, 2025 at 7:43 PM
hardcastlelab.bsky.social
Amazing results of gene therapy in very young children from @ucleye.bsky.social 👏👏 www.thelancet.com/journals/lan...
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...
www.thelancet.com
February 21, 2025 at 7:53 AM
petetheteapot.bsky.social
"[The] findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vision and evidence of some protection against progressive retinal degeneration [...]."
February 21, 2025 at 10:17 AM
stemcellreports.bsky.social
MAGIK: Human cone #photoreceptor transplantation in an AIPL1 model of end-stage Leber congenital amaurosis drives retinal remodeling & function, with light-evoked head-tracking behavior at physiologically relevant light levels
ow.ly/cVnA50Vq3nq

@isscr.org
March 27, 2025 at 7:59 PM
digital-health.bsky.social
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study

www.thelancet.com/journals/lan...

#RetinalDystrophy #AIPL1 #GeneTherapy #MedSky
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...
www.thelancet.com
February 27, 2025 at 1:38 PM
nplusodin.bsky.social
Медики из Королевского колледжа Лондона смогли вернуть зрение в одном глазу четырем слепым от рождения детям. В этом помог вирусный вектор, который доставил работающий ген в сетчатку

https://nplus1.ru/news/2025/02/21/gene-therapy-aipl1

#главноезанеделю
Генная терапия помогла слепым детям выполнить визуальные задания
N + 1 — главное издание о науке, технике и технологиях
nplus1.ru
February 23, 2025 at 5:14 PM
drtimos.bsky.social
Thoughts on this? >> Gene therapy can improve vision in young children with
AIPL1-associated retinal dystrophy - Medical Xpress: Gene therapy can improve vision in… >> Comment below! #strategy #competitiveintelligence #marketing #biotech #pharma #competitivemarketing #pharmaceutical #healthcare
Gene therapy can improve vision in young children with AIPL1-associated retinal dystrophy - Medical Xpress
Gene therapy can improve vision in young children with AIPL1-associated retinal dystrophy  Medical Xpress
dlvr.it
February 25, 2025 at 9:31 PM
dtake01.bsky.social
AAV delivery of AIPL1 resulted in sustained improvement in visual acuity in young children with AIPL1-associated retinal dystrophy

medicalxpress.com/news/2025-02...

www.thelancet.com/journals/lan...
Gene therapy can improve vision in young children with AIPL1-associated retinal dystrophy
Researchers from the NIHR Moorfields Biomedical Research Centre and University College London have found that gene therapy improved visual acuity and preserved retinal structure in young children with...
medicalxpress.com
February 26, 2025 at 4:01 AM
frostek.bsky.social
Jace Broadbin of Connecticut underwent successful Compassionate Use gene therapy in the UK for a form of Leber congenital amaurosis caused by a mutation in the AIPL1 gene.
UK Compassionate Use gene therapy for retinal disease improves vision for boy with LCA4, a form of Leber congenital amaurosis caused by a mutation in the AIPL1 gene.
hopeinfocus.org
May 1, 2025 at 4:25 PM
notreload.bsky.social
$MGTX +5% [Eli Lilly partners with MeiraGTx in a $475M deal for a breakthrough gene therapy, AAV-AIPL1, designed to restore vision in children born legally blind.] notreload.xyz/eli-lilly-me...
Eli Lilly, MeiraGTx Ink $475M Vision Gene Therapy Deal
Eli Lilly partners with MeiraGTx in a $475M deal for a breakthrough gene therapy, AAV-AIPL1, designed to restore vision in children born legally blind.
notreload.xyz
November 10, 2025 at 3:57 PM
tiggerfrank.bsky.social
Amazing gene therapy for retinal disease. The supplementary videos are interesting. www.thelancet.com/journals/lan...
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...
www.thelancet.com
February 21, 2025 at 12:47 PM
themestimes.bsky.social
Six-year-old Jace Broadbin, born blind due to Leber congenital amaurosis (LCA), a genetic condition caused by mutations in the AIPL1 gene, has regained partial vision through a pioneering gene therapy.

#GeneTherapy #MedicalBreakthrough #ChildhoodBlindness #LCA #VisionRestoration
April 30, 2025 at 6:03 PM