Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases

Identification of preexisting adaptive immunity to Cas9 proteins in humans

(2019) Carsten T. Charlesworth et al.   NATURE MEDICINE

Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges

(2019) Valentina Ghiaccio et al.   Molecular Diagnosis & Therapy

Disruptive Technology: CRISPR/Cas-Based Tools and Approaches

(2019) Petros Patsali et al.   Molecular Diagnosis & Therapy

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

(2018) Chiara Antoniani et al.   BLOOD

Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors

(2018) Silvana Konermann et al.   CELL

Evolving Industry Partnerships and Investments in Cell and Gene Therapies

(2018) Devyn M. Smith et al.   Cell Stem Cell

High-efficiency transduction of spinal cord motor neurons by intrauterine delivery of integration-deficient lentiviral vectors

(2018) Sherif G. Ahmed et al.   JOURNAL OF CONTROLLED RELEASE

Lysosomal acid lipase deficiency allograft recurrence and liver failure- clinical outcomes of 18 liver transplantation patients

(2018) Donna Lee Bernstein et al.   MOLECULAR GENETICS AND METABOLISM

In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation

(2018) Ana M. Moreno et al.   MOLECULAR THERAPY

Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

Fetal gene therapy for neurodegenerative disease of infants

(2018) Giulia Massaro et al.   NATURE MEDICINE

p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells

(2018) Robert J. Ihry et al.   NATURE MEDICINE

CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response

(2018) Emma Haapaniemi et al.   NATURE MEDICINE

Long-Term Hematopoietic Engraftment of Congenic Amniotic Fluid Stem Cells After in Utero Intraperitoneal Transplantation to Immune Competent Mice

(2018) Panicos Shangaris et al.   STEM CELLS AND DEVELOPMENT

Shwachman-Diamond Syndrome: Molecular Mechanisms and Current Perspectives

(2018) Valentino Bezzerri et al.   Molecular Diagnosis & Therapy

Innovative Therapies for Cystic Fibrosis: The Road from Treatment to Cure

(2018) Giulio Cabrini   Molecular Diagnosis & Therapy

CRISPR/Cas9-Mediated Correction of the FANCD1 Gene in Primary Patient Cells

(2017) Karolina Skvarova Kramarzova et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Cellular function reinstitution of offspring red blood cells cloned from the sickle cell disease patient blood post CRISPR genome editing

(2017) Jianguo Wen et al.   Journal of Hematology & Oncology

In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model

(2017) Citra N.Z. Mattar et al.   MOLECULAR THERAPY

CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease

(2017) Suk See De Ravin et al.   Science Translational Medicine

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

(2017) Giulia Schiroli et al.   Science Translational Medicine

Generation and characterization of a human iPSC cell line expressing inducible Cas9 in the “safe harbor” AAVS1 locus

(2017) Julio Castaño et al.   Stem Cell Research

Early Insights from Commercialization of Gene Therapies in Europe

(2017) Nicolas Touchot et al.   Genes

The prospects of CRISPR-based genome engineering in the treatment of neurodegenerative disorders

(2017) Jun Wan Shin et al.   Therapeutic Advances in Neurological Disorders

Clinical Practice Guidelines for Rare Diseases: The Orphanet Database

(2017) Sonia Pavan et al.   PLoS One

Somatic genome editing with CRISPR/Cas9 generates and corrects a metabolic disease

(2017) Kelsey E. Jarrett et al.   Scientific Reports

Genome editing with CRISPR/Cas9 in postnatal mice corrects PRKAG2 cardiac syndrome

(2016) Chang Xie et al.   CELL RESEARCH

Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9

(2016) Jun Wan Shin et al.   HUMAN MOLECULAR GENETICS

CRISPR-Mediated Base Editing without DNA Double-Strand Breaks

(2016) Brian S. Plosky   MOLECULAR CELL

In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa

(2016) Benjamin Bakondi et al.   MOLECULAR THERAPY

Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy

(2016) Jennifer E. Adair et al.   Nature Communications

Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia

(2016) Francis P. Pankowicz et al.   Nature Communications

Post-translational Regulation of Cas9 during G1 Enhances Homology-Directed Repair

(2016) Tony Gutschner et al.   Cell Reports

Transgene integration into the human AAVS1 locus enhances myosin II-dependent contractile force by reducing expression of myosin binding subunit 85

(2015) Takeomi Mizutani et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation

(2015) Xiaosong Huang et al.   STEM CELLS

Preparedness of the CTSA's Structural and Scientific Assets to Support the Mission of the National Center for Advancing Translational Sciences (NCATS)

(2012)   CTS-Clinical and Translational Science

European regulation on orphan medicinal products: 10 years of experience and future perspectives

(2011)   NATURE REVIEWS DRUG DISCOVERY

Hematopoietic Stem Cell Quiescence Promotes Error-Prone DNA Repair and Mutagenesis

(2010) Mary Mohrin et al.   Cell Stem Cell