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Induced Pluripotent Stem Cells Derived from Patients with CEP290-associated Ciliopathies and Unaffected Family Members
Summary: The National Eye Institute (NEI) seeks research collaborations and/or licensees for the use of iPS cells. Description of Technology: Approximately one-third of non-syndromic
retina
l dystrophies involve a defect in a ciliary protein. Non-syndromic retinal ciliopathies include retinitis pigmentosa, cone dystrophy, cone-rod dystrophy, macular...
Published: 4/24/2026
|
Updated: 8/2/2023
|
Inventor(s):
Anand Swaroop
,
Hiroko Shimada-Ishii
,
Yu Holly Chen
,
Milton English
Keywords(s):
Anosmia
,
Cell therapy
,
Ciliopathies
,
Congenital Blindness
,
Drug Development
,
GENE THERAPY
,
hearing loss
,
iPS
,
National Eye Institute
,
NEI
,
Pluripotent Stem Cells
,
Primary Cilia
,
Retina
,
Retina
l degeneration
,
Swaroop
,
VISION
Category(s):
Application > Research Materials
,
Collaboration Sought > Licensing
,
Collaboration Sought > Collaboration
,
TherapeuticArea > Ear, Nose, & Throat
,
TherapeuticArea > Ophthalmology
RP2 and RPGR Vectors For Treating X-linked Retinitis Pigmentosa
Abstract: X-linked forms of retinitis pigmentosa (XLRP) are relatively severe blinding disorders, resulting from progressive photoreceptor dysfunction primarily caused by mutations in RPGR or RP2 gene. This technology is poised to advance RPGR or RP2 gene therapy to clinical stage using AAV8 or AAV9 vector carrying human full-length RPGR or RP2-coding...
Published: 5/15/2025
|
Updated: 8/2/2023
|
Inventor(s):
Zhijian Wu
,
Anand Swaroop
,
Suddhasil Mookherjee
,
Suja Hiriyanna
,
Tiansen Li
Keywords(s):
GENE THERAPY
,
hereditary ocular disease
,
Pigmentosa
,
Retina
,
Wu
Category(s):
Collaboration Sought > Licensing
,
Collaboration Sought > Collaboration
,
Application > Therapeutics
,
TherapeuticArea > Ear, Nose, & Throat
,
TherapeuticArea > Ophthalmology
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