Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease

Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

(2022) Eman A. Taha et al.   JOURNAL OF CONTROLLED RELEASE

Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial

(2022) Elisa Magrin et al.   NATURE MEDICINE

Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin

(2022) Nithin Sam Ravi et al.   eLife

Interim Results of a Phase 1–2a Trial of Ad26.COV2.S Covid-19 Vaccine

(2021) Jerald Sadoff et al.   NEW ENGLAND JOURNAL OF MEDICINE

Hematopoietic Stem Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies

(2021) Claire M. Drysdale et al.   Cell Stem Cell

Plerixafor combined with G-CSF for stem cell mobilization in children qualified for autologous transplantation- single center experience

(2021) Iwona Malinowska et al.   TRANSFUSION AND APHERESIS SCIENCE

Expanding the Potential of Mammalian Genome Engineering via Targeted DNA Integration

(2021) Meng Zhang et al.   ACS Synthetic Biology

Virus-like particles: preparation, immunogenicity and their roles as nanovaccines and drug nanocarriers

(2021) Saghi Nooraei et al.   JOURNAL OF NANOBIOTECHNOLOGY

Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease

(2021) Naoya Uchida et al.   Molecular Therapy-Methods & Clinical Development

A pause in gene therapy: Reflecting on the unique challenges of sickle cell disease

(2021) Alexis Leonard et al.   MOLECULAR THERAPY

Rationally Designed Base Editors for Precise Editing of the Sickle Cell Disease Mutation

(2021) S. Haihua Chu et al.   CRISPR Journal

Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease

(2021) Alexis Leonard et al.   Blood Advances

Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species

(2021) Mohammadsharif Tabebordbar et al.   CELL

Base editing of haematopoietic stem cells rescues sickle cell disease in mice

(2021) Gregory A. Newby et al.   NATURE

Activation of γ-globin gene expression by GATA1 and NF-Y in hereditary persistence of fetal hemoglobin

(2021) Phillip A. Doerfler et al.   NATURE GENETICS

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

Lipid nanoparticles for mRNA delivery

(2021) Xucheng Hou et al.   Nature Reviews Materials

5-Azacytidine depletes hematopoietic stem cells and synergizes with an anti-CD117 antibody to augment donor engraftment in immunocompetent mice

(2021) Andriyana K. Bankova et al.   Blood Advances

Preliminary Safety and Efficacy Results from Precizn-1: An Ongoing Phase 1/2 Study on Zinc Finger Nuclease-Modified Autologous CD34+ HSPCs for Sickle Cell Disease (SCD)

(2021) Asif Alavi et al.   BLOOD

Clinical Results of the Drepaglobe Trial for Sickle Cell Disease Patients

(2021) Elisa Magrin et al.   BLOOD

Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

(2021) Mark C. Walters et al.   BLOOD

Cedar Trial in Progress: A First in Human, Phase 1/2 Study of the Correction of a Single Nucleotide Mutation in Autologous HSCs (GPH101) to Convert HbS to HbA for Treating Severe SCD

(2021) Julie Kanter et al.   BLOOD

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

(2021) John F. Tisdale et al.   BLOOD

CD117 Antibody Drug Conjugate-Based Conditioning Allows for Efficient Engraftment of Gene-Modified CD34+ Cells in a Rhesus Gene Therapy Model

(2021) Naoya Uchida et al.   BLOOD

Development of a quantitative prediction model for peripheral blood stem cell collection yield in the plerixafor era

(2021) Akira Ishii et al.   CYTOTHERAPY

Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease

(2021) Julie Kanter et al.   NEW ENGLAND JOURNAL OF MEDICINE

Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease

(2021) Sunita Goyal et al.   NEW ENGLAND JOURNAL OF MEDICINE

Comprehensive analysis of prime editing outcomes in human embryonic stem cells

(2021) Omer Habib et al.   NUCLEIC ACIDS RESEARCH

Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors

(2021) Chang Li et al.   Molecular Therapy-Methods & Clinical Development

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

(2020) Julie Kanter et al.   BLOOD

Baseline TP53 mutations in Adults with SCD developing Myeloid Malignancy following Hematopoietic Cell Transplantation

(2020) Jack Y Ghannam et al.   BLOOD

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

(2020) Leslie Weber et al.   Science Advances

A mutation-independent CRISPR-Cas9–mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency

(2020) Lili Wang et al.   Science Advances

Nanoparticles-mediated CRISPR/Cas9 delivery: Recent advances in cancer treatment

(2020) Shahin Aghamiri et al.   JOURNAL OF DRUG DELIVERY SCIENCE AND TECHNOLOGY

AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9

(2020) Ang Li et al.   MOLECULAR THERAPY

Correction of three prominent mutations in mouse and human models of Duchenne muscular dystrophy by single-cut genome editing

(2020) Yi-Li Min et al.   MOLECULAR THERAPY

Directed evolution of adenine base editors with increased activity and therapeutic application

(2020) Nicole M. Gaudelli et al.   NATURE BIOTECHNOLOGY

Therapeutic base editing of human hematopoietic stem cells

(2020) Jing Zeng et al.   NATURE MEDICINE

CRISPR off-target detection with DISCOVER-seq

(2020) Beeke Wienert et al.   Nature Protocols

Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants

(2020) Russell T. Walton et al.   SCIENCE

Viral nanoparticles for drug delivery, imaging, immunotherapy, and theranostic applications

(2020) Young Hun Chung et al.   ADVANCED DRUG DELIVERY REVIEWS

Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy

(2020) James M. Wilson et al.   HUMAN GENE THERAPY

Current Status and Challenges of DNA Base Editing Tools

(2020) You Kyeong Jeong et al.   MOLECULAR THERAPY

Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing

(2020) Tuo Wei et al.   Nature Communications

Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease

(2020) Matthew M. Hsieh et al.   Blood Advances

Delivery Approaches for Therapeutic Genome Editing and Challenges

(2020) Ilayda Ates et al.   Genes

A Review on the Synthesis and Functionalization of Gold Nanoparticles as a Drug Delivery Vehicle

(2020) Sundus Jabeen Amina et al.   International Journal of Nanomedicine

Safety and efficacy of the ChAdOx1 nCoV-19 vaccine (AZD1222) against SARS-CoV-2: an interim analysis of four randomised controlled trials in Brazil, South Africa, and the UK

(2020) Merryn Voysey et al.   LANCET

Efficacy and Safety of the mRNA-1273 SARS-CoV-2 Vaccine

(2020) Lindsey R. Baden et al.   NEW ENGLAND JOURNAL OF MEDICINE

Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine

(2020) Fernando P. Polack et al.   NEW ENGLAND JOURNAL OF MEDICINE

Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease

(2020) Erica B. Esrick et al.   NEW ENGLAND JOURNAL OF MEDICINE

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

Anti-human CD117 antibody-mediated bone marrow niche clearance in non-human primates and humanized NSG mice

(2019) Hye-Sook Kwon et al.   BLOOD

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

(2019) Sarah Marktel et al.   NATURE MEDICINE

Engineering of CRISPR-Cas12b for human genome editing

(2019) Jonathan Strecker et al.   Nature Communications

Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation

(2019) Agnieszka Czechowicz et al.   Nature Communications

Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges

(2019) Marina Cavazzana et al.   NATURE REVIEWS DRUG DISCOVERY

Highly efficient therapeutic gene editing of human hematopoietic stem cells

(2019) Yuxuan Wu et al.   NATURE MEDICINE

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

Bone marrow characterization in sickle cell disease: inflammation and stress erythropoiesis lead to suboptimal CD34 recovery

(2019) Alexis Leonard et al.   BRITISH JOURNAL OF HAEMATOLOGY

Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination

(2019) Renata M. Martin et al.   Cell Stem Cell

Long-term event-free survival, chimerism and fertility outcomes in 234 patients with sickle-cell anemia younger than 30 years after myeloablative conditioning and matched-sibling transplantation in France

(2019) Françoise Bernaudin et al.   HAEMATOLOGICA

Long-term ex vivo haematopoietic-stem-cell expansion allows nonconditioned transplantation

(2019) Adam C. Wilkinson et al.   NATURE

Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations

(2019) Reza Shahbazi et al.   NATURE MATERIALS

Gene therapy for primary immunodeficiency

(2019) Claire Booth et al.   HUMAN MOLECULAR GENETICS

Myeloid neoplasms in the setting of sickle cell disease: an intrinsic association with the underlying condition rather than a coincidence; report of 4 cases and review of the literature

(2019) Yang Li et al.   MODERN PATHOLOGY

Busulfan Combined with Immunosuppression Allows Efficient Engraftment of Gene-Modified Cells in a Rhesus Macaque Model

(2019) Naoya Uchida et al.   MOLECULAR THERAPY

High-Efficiency Lentiviral Transduction of Human CD34+ Cells in High-Density Culture with Poloxamer and Prostaglandin E2

(2019) Naoya Uchida et al.   Molecular Therapy-Methods & Clinical Development

Targeted Integration and High-Level Transgene Expression in AAVS1 Transgenic Mice after In Vivo HSC Transduction with HDAd5/35++ Vectors

(2019) Chang Li et al.   MOLECULAR THERAPY

Title: Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements

(2019) Richard A. Morgan et al.   MOLECULAR THERAPY

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

(2019) Janet L. Kwiatkowski et al.   BLOOD

In Vivo AAV-CRISPR/Cas9-Mediated Gene Editing Ameliorates Atherosclerosis in Familial Hypercholesterolemia

(2019) Huan Zhao et al.   CIRCULATION

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

High levels of AAV vector integration into CRISPR-induced DNA breaks

(2019) Killian S. Hanlon et al.   Nature Communications

Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders

(2019) Naoya Uchida et al.   Nature Communications

The deletion of mutant SOD1 via CRISPR/Cas9/sgRNA prolongs survival in an amyotrophic lateral sclerosis mouse model

(2019) Weisong Duan et al.   GENE THERAPY

Immunoresponse to Gene-Modified Hematopoietic Stem Cells

(2019) Claire M. Drysdale et al.   Molecular Therapy-Methods & Clinical Development

AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria

(2019) Daelyn Y. Richards et al.   Molecular Therapy-Methods & Clinical Development

Macrophage-Specific in Vivo Gene Editing Using Cationic Lipid-Assisted Polymeric Nanoparticles

(2018) Ying-Li Luo et al.   ACS Nano

Thermo-triggered Release of CRISPR-Cas9 System by Lipid-Encapsulated Gold Nanoparticles for Tumor Therapy

(2018) Peng Wang et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

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

(2018) Chiara Antoniani et al.   BLOOD

Direct Promoter Repression by BCL11A Controls the Fetal to Adult Hemoglobin Switch

(2018) Nan Liu et al.   CELL

Gene therapy for sickle cell disease: An update

(2018) Selami Demirci et al.   CYTOTHERAPY

Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice

(2018) Karine Sii-Felice et al.   EXPERIMENTAL HEMATOLOGY

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells

(2018) Joseph Long 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

Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding

(2018) Gabriella E. Martyn et al.   NATURE GENETICS

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

(2018) Alexis A. Thompson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nonviral gene editing via CRISPR/Cas9 delivery by membrane-disruptive and endosomolytic helical polypeptide

(2018) Hong-Xia Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Engineering the Delivery System for CRISPR-Based Genome Editing

(2018) Zachary Glass et al.   TRENDS IN BIOTECHNOLOGY

CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration

(2018) Taeyoung Koo et al.   Nature Communications

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

(2018) Jonathan D. Finn et al.   Cell Reports

VSV-G-Enveloped Vesicles for Traceless Delivery of CRISPR-Cas9

(2018) Claudia Montagna et al.   Molecular Therapy-Nucleic Acids

Sickle cell disease

(2018) Gregory J. Kato et al.   Nature Reviews Disease Primers

A Nonhuman Primate Transplantation Model to Evaluate Hematopoietic Stem Cell Gene Editing Strategies for β-Hemoglobinopathies

(2018) Olivier Humbert et al.   Molecular Therapy-Methods & Clinical Development

Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population

(2018) Vijaya L. Simhadri et al.   Molecular Therapy-Methods & Clinical Development

Integrating HDAd5/35++ Vectors as a New Platform for HSC Gene Therapy of Hemoglobinopathies

(2018) Chang Li et al.   Molecular Therapy-Methods & Clinical Development

Evolved Cas9 variants with broad PAM compatibility and high DNA specificity

(2018) Johnny H. Hu et al.   NATURE

An APOBEC3A-Cas9 base editor with minimized bystander and off-target activities

(2018) Jason M Gehrke et al.   NATURE BIOTECHNOLOGY

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

(2018) Christopher A. Vakulskas et al.   NATURE MEDICINE

Hydroxyurea in the management of sickle cell disease: pharmacogenomics and enzymatic metabolism

(2018) Sètondji Cocou Modeste Alexandre Yahouédéhou et al.   PHARMACOGENOMICS JOURNAL

Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major

(2018) Nikoletta Psatha et al.   Molecular Therapy-Methods & Clinical Development

Gesicle-Mediated Delivery of CRISPR/Cas9 Ribonucleoprotein Complex for Inactivating the HIV Provirus

(2018) Lee A. Campbell et al.   MOLECULAR THERAPY

Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair

(2018) Anastasia Lomova et al.   STEM CELLS

Genome editing in primary cells and in vivo using viral-derived Nanoblades loaded with Cas9-sgRNA ribonucleoproteins

(2018) Philippe E. Mangeot et al.   Nature Communications

Viral vector-based delivery of CRISPR/Cas9 and donor DNA for homology directed repair in an in vitro model for canine hemophilia B

(2018) Jian Gao et al.   Molecular Therapy-Nucleic Acids

Multistage Delivery Nanoparticle Facilitates Efficient CRISPR/dCas9 Activation and Tumor Growth Suppression In Vivo

(2018) Qi Liu et al.   Advanced Science

A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing

(2018) Ang Li et al.   Molecular Therapy-Methods & Clinical Development

Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications

(2017) Chang Liu et al.   JOURNAL OF CONTROLLED RELEASE

Mobilization of hematopoietic stem cells with the novel CXCR4 antagonist POL6326 (balixafortide) in healthy volunteers—results of a dose escalation trial

(2017) Darja Karpova et al.   Journal of Translational Medicine

Lipid Nanoparticle Systems for Enabling Gene Therapies

(2017) Pieter R. Cullis et al.   MOLECULAR THERAPY

CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

(2017) Alex Mas Monteys et al.   MOLECULAR THERAPY

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR–Cas9 nuclease off-targets

(2017) Shengdar Q Tsai et al.   NATURE METHODS

Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease

(2017) Kenneth I. Ataga et al.   NEW ENGLAND JOURNAL OF MEDICINE

In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni

(2017) Eunji Kim et al.   Nature Communications

Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice

(2017) Wenhan Yu et al.   Nature Communications

BLISS is a versatile and quantitative method for genome-wide profiling of DNA double-strand breaks

(2017) Winston X. Yan et al.   Nature Communications

Correction of β-thalassemia mutant by base editor in human embryos

(2017) Puping Liang et al.   Protein & Cell

In Vivo Knockout of the Vegfa Gene by Lentiviral Delivery of CRISPR/Cas9 in Mouse Retinal Pigment Epithelium Cells

(2017) Andreas Holmgaard et al.   Molecular Therapy-Nucleic Acids

Bone Marrow as a Hematopoietic Stem Cell Source for Gene Therapy in Sickle Cell Disease: Evidence from Rhesus and SCD Patients

(2017) Naoya Uchida et al.   Human Gene Therapy Clinical Development

Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C:G-to-T:A base editors with higher efficiency and product purity

(2017) Alexis C. Komor et al.   Science Advances

Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA

(2016) Jason B. Miller et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease

(2016) Mark C. Walters et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

(2016) Eliane Gluckman et al.   BLOOD

Nanoparticles and direct immunosuppression

(2016) Terrika A Ngobili et al.   EXPERIMENTAL BIOLOGY AND MEDICINE

Lentivirus pre-packed with Cas9 protein for safer gene editing

(2016) J G Choi et al.   GENE THERAPY

Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects

(2016) Jorge Mansilla-Soto et al.   HUMAN GENE THERAPY

The Role of Conditioning in Hematopoietic Stem-Cell Gene Therapy

(2016) Maria Ester Bernardo et al.   HUMAN GENE THERAPY

New agents in HSC mobilization

(2016) Mélanie J. Domingues et al.   INTERNATIONAL JOURNAL OF HEMATOLOGY

CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells

(2016) Megan D Hoban et al.   MOLECULAR THERAPY

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

(2016) Daniel P. Dever et al.   NATURE

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver et al.   NATURE

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

(2016) M. A. DeWitt et al.   Science Translational Medicine

Total body irradiation must be delivered at high dose for efficient engraftment and tolerance in a rhesus stem cell gene therapy model

(2016) Naoya Uchida et al.   Molecular Therapy-Methods & Clinical Development

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

(2015) M. D. Hoban et al.   BLOOD

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

A split-Cas9 architecture for inducible genome editing and transcription modulation

(2015) Bernd Zetsche et al.   NATURE BIOTECHNOLOGY

Functional footprinting of regulatory DNA

(2015) Jeff Vierstra et al.   NATURE METHODS

Development of an intein-mediated split–Cas9 system for gene therapy

(2015) Dong-Jiunn Jeffery Truong et al.   NUCLEIC ACIDS RESEARCH

Off-target Effects in CRISPR/Cas9-mediated Genome Engineering

(2015) Xiao-Hui Zhang et al.   Molecular Therapy-Nucleic Acids

Sickle cell disease: a neglected chronic disease of increasing global health importance

(2014) Subarna Chakravorty et al.   ARCHIVES OF DISEASE IN CHILDHOOD

Adenovirus: The First Effective In Vivo Gene Delivery Vector

(2014) Ronald G. Crystal   HUMAN GENE THERAPY

Nonmyeloablative HLA-Matched Sibling Allogeneic Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Phenotype

(2014) Matthew M. Hsieh et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Gold Nanoparticles for Nucleic Acid Delivery

(2014) Ya Ding et al.   MOLECULAR THERAPY

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

A guide to genome engineering with programmable nucleases

(2014) Hyongbum Kim et al.   NATURE REVIEWS GENETICS

Simple and Rapid In Vivo Generation of Chromosomal Rearrangements using CRISPR/Cas9 Technology

(2014) Rafael B. Blasco et al.   Cell Reports

Pre-existing immunity against Ad vectors

(2014) Hugues Fausther-Bovendo et al.   Human Vaccines & Immunotherapeutics

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

An Erythroid Enhancer of BCL11A Subject to Genetic Variation Determines Fetal Hemoglobin Level

(2013) D. E. Bauer et al.   SCIENCE

Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo

(2012) Muthusamy Jayaraman et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

(2012) G. Buchlis et al.   BLOOD

Directed evolution of novel adeno-associated viruses for therapeutic gene delivery

(2012) M A Bartel et al.   GENE THERAPY

Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo

(2012) N A McNeer et al.   GENE THERAPY

Impact of Preexisting Adenovirus Vector Immunity on Immunogenicity and Protection Conferred with an Adenovirus-Based H5N1 Influenza Vaccine

(2012) Aseem Pandey et al.   PLoS One

Optimal conditions for lentiviral transduction of engrafting human CD34+ cells

(2011) N Uchida et al.   GENE THERAPY

Nanoparticle PEGylation for imaging and therapy

(2011) Jesse V Jokerst et al.   Nanomedicine

Correction of Sickle Cell Disease in Adult Mice by Interference with Fetal Hemoglobin Silencing

(2011) J. Xu et al.   SCIENCE

Update on fetal hemoglobin gene regulation in hemoglobinopathies

(2010) Daniel E Bauer et al.   CURRENT OPINION IN PEDIATRICS

Chicken HS4 Insulators Have Minimal Barrier Function Among Progeny of Human Hematopoietic Cells Transduced With an HIV1-based Lentiviral Vector

(2010) Naoya Uchida et al.   MOLECULAR THERAPY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Defining the Intensity of Conditioning Regimens: Working Definitions

(2009) Andrea Bacigalupo et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Integration-deficient Lentiviral Vectors: A Slow Coming of Age

(2009) Klaus Wanisch et al.   MOLECULAR THERAPY

Allogeneic Hematopoietic Stem-Cell Transplantation for Sickle Cell Disease

(2009) Matthew M. Hsieh et al.   NEW ENGLAND JOURNAL OF MEDICINE

Rapid mobilization of functional donor hematopoietic cells without G-CSF using AMD3100, an antagonist of the CXCR4/SDF-1 interaction

(2008) S. M. Devine et al.   BLOOD

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION

Probing cells with noble metal nanoparticle aggregates

(2008) Maryuri Roca et al.   Nanomedicine

In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of  -thalassemia

(2008) A. Miccio et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Human Fetal Hemoglobin Expression Is Regulated by the Developmental Stage-Specific RepressorBCL11A

(2008) Vijay G. Sankaran et al.   SCIENCE