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Search Results - lca
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Gene Therapy for Treatment of CRX-Autosomal Dominant Retinopathies
Abstract: Mutations in the cone rod homeobox (CRX) transcription factor lead to distinct retinopathy phenotypes, including early-onset vision impairment in dominant Leber congenital amaurosis (
LCA
). Adeno-Associated virus (AAV) vector-mediated delivery of a CRX cDNA under the control of a CRX promoter region partially restored photoreceptor phenotype...
Published: 12/6/2024
|
Inventor(s):
Anand Swaroop
,
Kamil Kruczek
,
Suja Hiriyanna
,
Zhijian Wu
Keywords(s):
AAV
,
Adeno-associated Virus
,
Cone-rod Dystrophy
,
CRD
,
GENE THERAPY
,
LCA
,
Leber Congenital Amaurosis
,
Lentivirus
,
NEI
,
rare disease
,
Retinitis Pigmentosa
,
Retinopathies
,
RP
,
Swaroop
Category(s):
Collaboration Sought > Licensing
,
Application > Therapeutics
,
Collaboration Sought > Collaboration
,
TherapeuticArea > Ear, Nose, & Throat
,
TherapeuticArea > Ophthalmology