In utero gene therapy rescues vision in a murine model of congenital blindness.

[1]  B. Jones,et al.  Retinal remodeling triggered by photoreceptor degenerations , 2003, The Journal of comparative neurology.

[2]  E. Pugh,et al.  Delivery of Adeno-Associated Virus Vectors to the Fetal Retina: Impact of Viral Capsid Proteins on Retinal Neuronal Progenitor Transduction , 2003, Journal of Virology.

[3]  Edwin M Stone,et al.  Crumbs homolog 1 (CRB1) mutations result in a thick human retina with abnormal lamination. , 2003, Human molecular genetics.

[4]  M. Seeliger,et al.  Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog. , 2003, Investigative ophthalmology & visual science.

[5]  Charles Coutelle,et al.  Ultrasound-guided percutaneous delivery of adenoviral vectors encoding the beta-galactosidase and human factor IX genes to early gestation fetal sheep in utero. , 2003, Human gene therapy.

[6]  M. Dallman,et al.  In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor. , 2003, Blood.

[7]  Jian-xing Ma,et al.  Correlation of regenerable opsin with rod ERG signal in Rpe65-/- mice during development and aging. , 2002, Investigative ophthalmology & visual science.

[8]  M. R. Hough,et al.  In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression. , 2002, Human gene therapy.

[9]  T. Shirasawa,et al.  Adenoviral expression of protein‐L‐isoaspartyl methyltransferase (PIMT) partially attenuates the biochemical changes in PIMT‐deficient mice , 2002, Journal of neuroscience research.

[10]  S. Hanein,et al.  Prenatal human ocular degeneration occurs in Leber's congenital amaurosis (LCA2) , 2002, The journal of gene medicine.

[11]  A. D. den Hollander,et al.  Molecular genetics of Leber congenital amaurosis. , 2002, Human molecular genetics.

[12]  Herbert Jägle,et al.  Reorganization of human cortical maps caused by inherited photoreceptor abnormalities , 2002, Nature Neuroscience.

[13]  R. Weleber Infantile and childhood retinal blindness: A molecular perspective (The Franceschetti Lecture) , 2002, Ophthalmic genetics.

[14]  S. S. Case,et al.  Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): lung-targeting approaches. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.

[15]  A. V. Cideciyan,et al.  Augmented rod bipolar cell function in partial receptor loss: an ERG study in P23H rhodopsin transgenic and aging normal rats , 2001, Vision Research.

[16]  Lili Wang,et al.  Hybrid Vectors Based on Adeno-Associated Virus Serotypes 2 and 5 for Muscle-Directed Gene Transfer , 2001, Journal of Virology.

[17]  Jean Bennett,et al.  Gene therapy restores vision in a canine model of childhood blindness , 2001, Nature Genetics.

[18]  B. Lorenz,et al.  Early-onset severe rod-cone dystrophy in young children with RPE65 mutations. , 2000, Investigative ophthalmology & visual science.

[19]  T. Aleman,et al.  Rapid restoration of visual pigment and function with oral retinoid in a mouse model of childhood blindness. , 2000, Proceedings of the National Academy of Sciences of the United States of America.

[20]  H. Kazazian,et al.  Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero. , 1999, Proceedings of the National Academy of Sciences of the United States of America.

[21]  T. Aleman,et al.  Retinal Rod Photoreceptor–Specific Gene Mutation Perturbs Cone Pathway Development , 1999, Neuron.

[22]  D. Bok,et al.  Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle , 1998, Nature Genetics.

[23]  W. Anderson,et al.  In utero gene therapy: transfer and long-term expression of the bacterial neo(r) gene in sheep after direct injection of retroviral vectors into preimmune fetuses. , 1998, Human gene therapy.

[24]  A. Fulton,et al.  Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or leber congenital amaurosis. , 1998, Proceedings of the National Academy of Sciences of the United States of America.

[25]  C. Hamel,et al.  Rat messenger RNA for the retinal pigment epithelium‐specific protein RPE65 gradually accumulates in two weeks from late embryonic days 1 , 1998, FEBS letters.

[26]  E. Zrenner,et al.  Mutations in RPE65 cause Leber's congenital amaurosis , 1997, Nature Genetics.

[27]  A. Cideciyan,et al.  An Alternative Phototransduction Model for Human Rod and Cone ERG a-waves: Normal Parameters and Variation with Age , 1996, Vision Research.

[28]  H. Sekhon,et al.  In utero gene transfer into the pulmonary epithelium , 1995, Nature Medicine.

[29]  K. Nakanishi,et al.  Isorhodopsin II: artificial photosensitive pigment formed from 9,13-dicis retinal. , 1975, Proceedings of the National Academy of Sciences of the United States of America.

[30]  J. Bennett,et al.  Gene therapy and animal models for retinal disease. , 2003, Developments in ophthalmology.

[31]  W. Rascher,et al.  Sustained delivery of therapeutic concentrations of human clotting factor IX – a comparison of adenoviral and AAV vectors administered in utero , 2002, The journal of gene medicine.