دورية أكاديمية
Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer.
| العنوان: | Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer. |
|---|---|
| المؤلفون: | Takahashi M; Laboratory of Genetics, The Salk Institute for Biological Studies, La Jolla, California 92037, USA., Miyoshi H, Verma IM, Gage FH |
| المصدر: | Journal of virology [J Virol] 1999 Sep; Vol. 73 (9), pp. 7812-6. |
| نوع المنشور: | Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, P.H.S. |
| اللغة: | English |
| بيانات الدورية: | Publisher: American Society For Microbiology Country of Publication: United States NLM ID: 0113724 Publication Model: Print Cited Medium: Print ISSN: 0022-538X (Print) Linking ISSN: 0022538X NLM ISO Abbreviation: J Virol Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: Washington Dc : American Society For Microbiology Original Publication: Baltimore, American Society for Microbiology. |
| مواضيع طبية MeSH: | Gene Transfer Techniques* , Genetic Vectors* , HIV-1* , Phosphoric Diester Hydrolases* , Retinal Rod Photoreceptor Cells*, 3',5'-Cyclic-GMP Phosphodiesterases/*genetics , Retinitis Pigmentosa/*therapy, Animals ; Cattle ; Cyclic Nucleotide Phosphodiesterases, Type 6 ; Genetic Therapy ; Humans ; Mice ; Mice, Inbred C3H |
| مستخلص: | Retinitis pigmentosa (RP) is the most common inherited retinal disease, in which photoreceptor cells degenerate, leading to blindness. Mutations in the rod photoreceptor cGMP phosphodiesterase beta subunit (PDEbeta) gene are found in patients with autosomal recessive RP as well as in the rd mouse. We have recently shown that lentivirus vectors based on human immunodeficiency virus (HIV) type 1 achieve stable and efficient gene transfer into retinal cells. In this study, we evaluated the potential of HIV vector-mediated gene therapy for RP in the rd mouse. HIV vectors containing a gene encoding a hemagglutinin (HA)-tagged PDEbeta were injected into the subretinal spaces of newborn rd mouse eyes. One to three rows of photoreceptor nuclei were observed in the eyes for at least 24 weeks postinjection, whereas no photoreceptor cells remained in the eyes of control animals at 6 weeks postinjection. Expression of HA-tagged PDEbeta in the rescued photoreceptor cells was confirmed by two-color confocal immunofluorescence analysis using anti-HA and anti-opsin antibodies. HIV vector-mediated gene therapy appears to be a promising means for the treatment of recessive forms of inherited retinal degeneration. |
| References: | Proc Natl Acad Sci U S A. 1995 Apr 11;92(8):3249-53. (PMID: 7724547) Exp Eye Res. 1994 Apr;58(4):397-408. (PMID: 7925677) Genomics. 1995 Nov 1;30(1):1-7. (PMID: 8595886) Science. 1996 Apr 12;272(5259):263-7. (PMID: 8602510) Nat Med. 1996 Jun;2(6):649-54. (PMID: 8640555) Proc Natl Acad Sci U S A. 1996 Oct 15;93(21):11382-8. (PMID: 8876144) Mol Med Today. 1996 Sep;2(9):380-6. (PMID: 8885257) Hum Gene Ther. 1997 Mar 1;8(4):423-30. (PMID: 9054517) Proc Natl Acad Sci U S A. 1997 Jun 24;94(13):6916-21. (PMID: 9192666) J Virol. 1997 Sep;71(9):6641-9. (PMID: 9261386) Gene Ther. 1997 Jul;4(7):683-90. (PMID: 9282169) Proc Natl Acad Sci U S A. 1997 Sep 16;94(19):10319-23. (PMID: 9294208) Nat Biotechnol. 1997 Sep;15(9):871-5. (PMID: 9306402) Proc Natl Acad Sci U S A. 1998 Sep 15;95(19):11377-82. (PMID: 9736744) J Virol. 1998 Nov;72(11):8463-71. (PMID: 9765382) Hum Mol Genet. 1998 Nov;7(12):1893-900. (PMID: 9811932) J Virol. 1998 Dec;72(12):9873-80. (PMID: 9811723) J Virol. 1999 Jan;73(1):576-84. (PMID: 9847362) Nat Genet. 1997 Nov;17(3):314-7. (PMID: 9354796) Invest Ophthalmol Vis Sci. 1997 Dec;38(13):2857-63. (PMID: 9418740) J Virol. 1998 Jan;72(1):811-6. (PMID: 9420292) Hum Gene Ther. 1998 Jan 1;9(1):81-6. (PMID: 9458245) Nat Med. 1998 Aug;4(8):967-71. (PMID: 9701253) J Virol. 1998 Oct;72(10):8150-7. (PMID: 9733856) Am J Anat. 1972 Feb;133(2):179-212. (PMID: 5009246) Invest Ophthalmol Vis Sci. 1977 Oct;16(10):947-62. (PMID: 908648) Invest Ophthalmol Vis Sci. 1978 Jun;17(6):489-98. (PMID: 659071) Surv Ophthalmol. 1988 Nov-Dec;33(3):137-77. (PMID: 3068820) FEBS Lett. 1991 Jan 14;278(1):107-14. (PMID: 1847109) Proc Natl Acad Sci U S A. 1991 Oct 1;88(19):8322-6. (PMID: 1656438) Proc Natl Acad Sci U S A. 1992 May 15;89(10):4422-6. (PMID: 1350091) Proc Natl Acad Sci U S A. 1993 May 1;90(9):3968-72. (PMID: 8387203) Invest Ophthalmol Vis Sci. 1994 Apr;35(5):2535-42. (PMID: 8163342) Invest Ophthalmol Vis Sci. 1994 Apr;35(5):2543-9. (PMID: 8163343) Hum Mol Genet. 1995;4 Spec No:1739-43. (PMID: 8541873) |
| المشرفين على المادة: | EC 3.1.4.- (Phosphoric Diester Hydrolases) EC 3.1.4.35 (3',5'-Cyclic-GMP Phosphodiesterases) EC 3.1.4.35 (Cyclic Nucleotide Phosphodiesterases, Type 6) EC 3.1.4.35 (PDE6B protein, human) EC 3.1.4.35 (Pde6b protein, mouse) |
| تواريخ الأحداث: | Date Created: 19990810 Date Completed: 19990907 Latest Revision: 20200724 |
| رمز التحديث: | 20240627 |
| مُعرف محوري في PubMed: | PMC104309 |
| DOI: | 10.1128/JVI.73.9.7812-7816.1999 |
| PMID: | 10438872 |
| قاعدة البيانات: | MEDLINE |
كن أول من يترك تعليقا!