AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models

CHM/REP1 cDNA delivery by lentiviral vectors provides functional expression of the transgene in the retinal pigment epithelium of choroideremia mice

(2012) Tanya Tolmachova et al.   JOURNAL OF GENE MEDICINE

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

(2012) J. Bennett et al.   Science Translational Medicine

Choroideremia: Effect of age on visual acuity in patients and female carriers

(2011) Razek Georges Coussa et al.   OPHTHALMIC GENETICS

Choroideremia: A review of general findings and pathogenesis

(2011) Razek Georges Coussa et al.   OPHTHALMIC GENETICS

Rescue of Photoreceptor Degeneration by Curcumin in Transgenic Rats with P23H Rhodopsin Mutation

(2011) Vidyullatha Vasireddy et al.   PLoS One

When Fibroblasts MET iPSCs

(2010) Jose M. Polo et al.   Cell Stem Cell

Highly Efficient Reprogramming to Pluripotency and Directed Differentiation of Human Cells with Synthetic Modified mRNA

(2010) Luigi Warren et al.   Cell Stem Cell

Epigenetic memory in induced pluripotent stem cells

(2010) K. Kim et al.   NATURE

Cell type of origin influences the molecular and functional properties of mouse induced pluripotent stem cells

(2010) Jose M Polo et al.   NATURE BIOTECHNOLOGY

Generation of Transgene-Free Lung Disease-Specific Human Induced Pluripotent Stem Cells Using a Single Excisable Lentiviral Stem Cell Cassette

(2010) Aba Somers et al.   STEM CELLS

CHMgene molecular analysis and X-chromosome inactivation pattern determination in two families with choroideremia

(2009) Hector J. Perez-Cano et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

(2009) Albert M Maguire et al.   LANCET

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

(2009) Francesca Simonelli et al.   MOLECULAR THERAPY

The functional effect of pathogenic mutations in Rab escort protein 1

(2009) Y.V. Sergeev et al.   MUTATION RESEARCH-FUNDAMENTAL AND MOLECULAR MECHANISMS OF MUTAGENESIS

Loss-of-Function Mutations in Rab Escort Protein 1 (REP-1) Affect Intracellular Transport in Fibroblasts and Monocytes of Choroideremia Patients

(2009) Natalia V. Strunnikova et al.   PLoS One

Modeling early retinal development with human embryonic and induced pluripotent stem cells

(2009) J. S. Meyer et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Choroideremia: New Findings from Ocular Pathology and Review of Recent Literature

(2009) Ian M. MacDonald et al.   SURVEY OF OPHTHALMOLOGY

Multispectral imaging of hematopoietic cells: Where flow meets morphology

(2008) Kathleen E. McGrath et al.   JOURNAL OF IMMUNOLOGICAL METHODS

Human embryonic stem cell derivation from poor-quality embryos

(2008) Paul H Lerou et al.   NATURE BIOTECHNOLOGY

Derivation and maintenance of human embryonic stem cells from poor-quality in vitro fertilization embryos

(2008) Paul H Lerou et al.   Nature Protocols

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE