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Gene Therapy for Retinal Disease

Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania, U.S.A. [Pg.157]

Vector Nucleic acid Cargo size limit Target cells Stability Immune response Cellular receptor Intake protein [Pg.158]

Lipofection/liposomes (2) DNA/RNA/ liposomes/(Sendai virus) No limit NR NR NR [Pg.158]

Immunoliposomes (3) DNA + pegylated liposomes targeted to the transferrin receptor No limit Ciliary body, iris, corneal epithelium 48 hr NR Transferrin receptor NR [Pg.158]

Electroporation Gene gun DNA/RNA DNA No limit No limit Ganglion cells (4,5) Corneal epithelium (6) 1 wk NR NR NA NA [Pg.158]


Recent efforts have focused on using these highly regulated gene expression systems in gene therapies for disease. Applications include modification of stem cells (Moutsatsos et al., 2001), treatment of retinal diseases (Dejneka et al., 2001) and immunotherapy for cancer (Nakagawa et al., 2001). [Pg.22]


See other pages where Gene Therapy for Retinal Disease is mentioned: [Pg.157]    [Pg.159]    [Pg.161]    [Pg.161]    [Pg.163]    [Pg.165]    [Pg.167]    [Pg.169]    [Pg.171]    [Pg.173]    [Pg.157]    [Pg.159]    [Pg.161]    [Pg.161]    [Pg.163]    [Pg.165]    [Pg.167]    [Pg.169]    [Pg.171]    [Pg.173]    [Pg.113]    [Pg.85]    [Pg.34]    [Pg.107]    [Pg.161]    [Pg.162]    [Pg.164]    [Pg.165]    [Pg.165]    [Pg.167]    [Pg.169]    [Pg.402]    [Pg.243]    [Pg.166]    [Pg.335]    [Pg.810]    [Pg.283]    [Pg.163]   


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