相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene
Daniel C. Chung et al.
AMERICAN JOURNAL OF OPHTHALMOLOGY (2019)
Current Treatment Options in NeurologySMA Therapeutics
Megan A. Waldrop et al.
CURRENT TREATMENT OPTIONS IN NEUROLOGY (2019)
Gene therapy and the adeno-associated virus in the treatment of genetic and acquired ophthalmic diseases in humans: Trials, future directions and safety considerations
Charmaine A. Ramlogan-Steel et al.
CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY (2019)
Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy
Samiah Al-Zaidy et al.
PEDIATRIC PULMONOLOGY (2019)
Transthyretin familial amyloid polyneuropathy: an update
Violaine Plante-Bordeneuve
JOURNAL OF NEUROLOGY (2018)
Axicabtagene ciloleucel, a first-in-class CAR T cell therapy for aggressive NHL
Zachary J. Roberts et al.
LEUKEMIA & LYMPHOMA (2018)
Transthyretin familial amyloid polyneuropathy (TTR-FAP): Parameters for early diagnosis
Fabiola Escolano-Lozano et al.
BRAIN AND BEHAVIOR (2018)
Oncorine, the World First Oncolytic Virus Medicine and its Update in China
Min Liang
CURRENT CANCER DRUG TARGETS (2018)
A guide to manufacturing CAR T cell therapies
Philipp Vormittag et al.
CURRENT OPINION IN BIOTECHNOLOGY (2018)
Pegaptanib: choroidal neovascularization in patients with age-related macular degeneration and previous arterial thromboembolic events
Maurizio Battaglia Parodi et al.
EUROPEAN JOURNAL OF OPHTHALMOLOGY (2018)
CAR-T: trailblazing the path from clinical development to the clinic
Samuele Butera
GENE THERAPY (2018)
The First Approved Gene Therapy Product for Cancer Ad-p53 (Gendicine): 12 Years in the Clinic
Wei-Wei Zhang et al.
HUMAN GENE THERAPY (2018)
Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety
Maria Pia Cicalese et al.
MOLECULAR THERAPY (2018)
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
D. Adams et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia
S. L. Maude et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
INVOSSA, A FIRST-IN-CLASS OF CELL AND GENE THERAPY FOR OSTEOARTHRITIS TREATMENT: THE PHASE III TRIAL
B. Lee
OSTEOARTHRITIS AND CARTILAGE (2018)
INVOSSA-K INDUCES AN ANTI-INFLAMMATORY ENVIRONMENT IN A RAT MIA MODEL VIA MACROPHAGE POLARIZATION
H. Lee et al.
OSTEOARTHRITIS AND CARTILAGE (2018)
Molecular genetics and emerging therapies for retinitis pigmentosa: Basic research and clinical perspectives
Marina Franca Dias et al.
PROGRESS IN RETINAL AND EYE RESEARCH (2018)
Gene therapy: evidence, value and affordability in the US health care system
Grace Hampson et al.
JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH (2018)
Emerging Issues in AAV-Mediated In Vivo Gene Therapy
Pasqualina Colella et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)
FDA approves patisiran to treat hereditary transthyretin amyloidosis
Heather Wood
Nature Reviews Neurology (2018)
Voretigene neparvovec-rzyl for the treatment of biallelic RPE65 mutation-associated retinal dystrophy
Stephen Russell et al.
EXPERT OPINION ON ORPHAN DRUGS (2018)
Gene therapy for RPE65-related retinal disease
Virginia Miraldi Utz et al.
OPHTHALMIC GENETICS (2018)
Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease
Vamshi K. Rao et al.
JOURNAL OF MANAGED CARE & SPECIALTY PHARMACY (2018)
Talimogene Laherparepvec (T-VEC) and Other Oncolytic Viruses for the Treatment of Melanoma
Praveen K. Bommareddy et al.
AMERICAN JOURNAL OF CLINICAL DERMATOLOGY (2017)
Efficacy and safety of nusinersen in infants with presymptomatic spinal muscular atrophy (SMA): Interim results from the NURTURE study
E. Bertini et al.
EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY (2017)
AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones
Jerry R. Mendell et al.
EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY (2017)
Nusinersen for the treatment of spinal muscular atrophy
Claudia A. Chiriboga
EXPERT REVIEW OF NEUROTHERAPEUTICS (2017)
Spinraza-a rare disease success story
V. Prakash
GENE THERAPY (2017)
Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug
Francesca Ferrua et al.
HUMAN GENE THERAPY (2017)
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Stephen Russell et al.
LANCET (2017)
Viral vanguard: Designing cancer-killing viruses to chase metastatic tumors
Shraddha Chakradhar
NATURE MEDICINE (2017)
Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy
David R. Corey
NATURE NEUROSCIENCE (2017)
2016 EMA drug approval recommendations
Asher Mullard
NATURE REVIEWS DRUG DISCOVERY (2017)
Efficacy and safety of nusinersen in children with later-onset spinal muscular atrophy (SMA): end of study results from the phase 3 CHERISH study
E. Mercuri et al.
NEUROMUSCULAR DISORDERS (2017)
Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma
S. S. Neelapu et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
INVOSSA™ (TISSUEGENE-C) INDUCES AN ANTI-INFLAMMATORY ENVIRONMENT IN THE ARTHRITIC KNEE JOINTS VIA MACROPHAGE POLARIZATION
K. Choi et al.
OSTEOARTHRITIS AND CARTILAGE (2017)
Chimeric antigen receptor T-cell therapy for glioblastoma
Analiz Rodriguez et al.
TRANSLATIONAL RESEARCH (2017)
Acute lymphoblastic leukemia: a comprehensive review and 2017 update
T. Terwilliger et al.
BLOOD CANCER JOURNAL (2017)
Eteplirsen in the treatment of Duchenne muscular dystrophy
Kenji Rowel Q. Lim et al.
DRUG DESIGN DEVELOPMENT AND THERAPY (2017)
Nusinersen for the treatment of spinal muscular atrophy
Claudia A. Chiriboga
EXPERT REVIEW OF NEUROTHERAPEUTICS (2017)
The budget impact and cost-effectiveness of defibrotide for treatment of veno-occlusive disease with multi-organ dysfunction in patients post-hematopoietic stem cell transplant
David L. Veenstra et al.
JOURNAL OF MEDICAL ECONOMICS (2017)
A mouse anti-myostatin antibody increases muscle mass and improves muscle strength and contractility in the mdx mouse model of Duchenne muscular dystrophy and its humanized equivalent, domagrozumab (PF-06252616), increases muscle volume in cynomolgus monkeys
Michael St. Andre et al.
SKELETAL MUSCLE (2017)
CRISPR/Cas9 delivery with one single adenoviral vector devoid of all viral genes
Eric Ehrke-Schulz et al.
SCIENTIFIC REPORTS (2017)
Clinical and biologic covariates of outcomes in ZUMA-1: A pivotal trial of axicabtagene ciloleucel (axi-cel; KTE-C19) in patients with refractory aggressive non-Hodgkin lymphoma (r-NHL).
Frederick Lundry Locke et al.
JOURNAL OF CLINICAL ONCOLOGY (2017)
Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy
David Adams et al.
BMC NEUROLOGY (2017)
Nusinersen: The First Option Beyond Supportive Care for Spinal Muscular Atrophy
Vikas Maharshi et al.
CLINICAL DRUG INVESTIGATION (2017)
Global Pivotal Phase 2 Trial of the CD19-Targeted Therapy CTL019 In Adult Patients with Relapsed or Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL)-An Interim Analysis
Stephen Schuster et al.
CLINICAL LYMPHOMA MYELOMA & LEUKEMIA (2017)
Clinical development of CAR T cells-challenges and opportunities in translating innovative treatment concepts
Jessica Hartmann et al.
EMBO MOLECULAR MEDICINE (2017)
Results of an International Postmarketing Surveillance Study of pl-VEGF165 Safety and Efficacy in 210 Patients with Peripheral Arterial Disease
Roman Deev et al.
AMERICAN JOURNAL OF CARDIOVASCULAR DRUGS (2017)
Adenovirus-mediated gene delivery: Potential applications for gene and cell-based therapies in the new era of personalized medicine
Cody S. Lee et al.
GENES & DISEASES (2017)
The impact of HLA matching on outcomes of unmanipulated haploidentical HSCT is modulated by GVHD prophylaxis
Francesca Lorentino et al.
BLOOD ADVANCES (2017)
Viral Vectors, Engineered Cells and the CRISPR Revolution
James E. DiCarlo et al.
PRECISION MEDICINE, CRISPR, AND GENOME ENGINEERING: MOVING FROM ASSOCIATION TO BIOLOGY AND THERAPEUTICS (2017)
Molecular Pathways: Mechanism of Action for Talimogene Laherparepvec, a New Oncolytic Virus Immunotherapy
Frederick J. Kohlhapp et al.
CLINICAL CANCER RESEARCH (2016)
Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis
James S. Butler et al.
AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS (2016)
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2016)
Defibrotide for Treatment of Severe Veno-Occlusive Disease in Pediatrics and Adults: An Exploratory Analysis Using Data from the Center for International Blood and Marrow Transplant Research
Christopher Strouse et al.
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2016)
Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure
Paul G. Richardson et al.
BLOOD (2016)
Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency
Maria Pia Cicalese et al.
BLOOD (2016)
Oncolytic virus therapy: A new era of cancer treatment at dawn
Hiroshi Fukuhara et al.
CANCER SCIENCE (2016)
Current and Future Treatment Approaches in Transthyretin Familial Amyloid Polyneuropathy
Philippe Kerschen et al.
CURRENT TREATMENT OPTIONS IN NEUROLOGY (2016)
TTR kinetic stabilizers and TTR gene silencing: a new era in therapy for familial amyloidotic polyneuropathies
David Adams et al.
EXPERT OPINION ON PHARMACOTHERAPY (2016)
How to select the best available related or unrelated donor of hematopoietic stem cells?
Jean-Marie Tiercy
HAEMATOLOGICA (2016)
Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreatitis
Daniel Gaudet et al.
HUMAN GENE THERAPY (2016)
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
Richard S. Finkel et al.
LANCET (2016)
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial
Jean Bennett et al.
LANCET (2016)
Considerations and challenges for patients with refractory and relapsed acute myeloid leukaemia
Jonathan Kell
LEUKEMIA RESEARCH (2016)
Eteplirsen Approved for Duchenne Muscular Dystrophy: The FDA Faces a Difficult Choice
Cy A. Stein
MOLECULAR THERAPY (2016)
Big win possible for Ionis/Biogen antisense drug in muscular atrophy
Ken Garber
NATURE BIOTECHNOLOGY (2016)
Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials
Eugenio Mercuri et al.
NEUROMUSCULAR DISORDERS (2016)
How to select the best available related or unrelated donor of hematopoietic stem cells?
Jean-Marie Tiercy
HAEMATOLOGICA (2016)
Adenosine Deaminase Deficiency - More Than Just an Immunodeficiency
Kathryn V. Whitmore et al.
FRONTIERS IN IMMUNOLOGY (2016)
Clinical development of gene therapy: results and lessons from recent successes
Sandeep R. P. Kumar et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2016)
Aflibercept for neovascular age-related macular degeneration
Salman Sarwar et al.
COCHRANE DATABASE OF SYSTEMATIC REVIEWS (2016)
Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides
Richard S. Geary et al.
ADVANCED DRUG DELIVERY REVIEWS (2015)
Exon skipping therapy for Duchenne muscular dystrophy
Ryszard Kole et al.
ADVANCED DRUG DELIVERY REVIEWS (2015)
Mipomersen, an Antisense Oligonucleotide to Apolipoprotein B-100, Reduces Lipoprotein(a) in Various Populations With Hypercholesterolemia Results of 4 Phase III Trials
Raul D. Santos et al.
ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY (2015)
Haploidentical HSCT: a 15-year experience at San Raffaele
C. Bonini et al.
BONE MARROW TRANSPLANTATION (2015)
Sinusoidal obstruction syndrome/veno-occlusive disease: current situation and perspectives-a position statement from the European Society for Blood and Marrow Transplantation (EBMT)
M. Mohty et al.
BONE MARROW TRANSPLANTATION (2015)
Intracellular delivery of VEGF165 encoding gene therapeutic using trifunctional copolymers of ethylene oxide and propylene oxide
Oksana Bondar et al.
EUROPEAN POLYMER JOURNAL (2015)
TRUCKs: the fourth generation of CARs
Markus Chmielewski et al.
EXPERT OPINION ON BIOLOGICAL THERAPY (2015)
Adoptive therapy with CAR redirected T cells: the challenges in targeting solid tumors
Hinrich Abken
IMMUNOTHERAPY (2015)
Talimogene laherparepvec (T-VEC) for the treatment of advanced melanoma
Douglas B. Johnson et al.
IMMUNOTHERAPY (2015)
pCMV-vegf165 Intramuscular Gene Transfer is an Effective Method of Treatment for Patients With Chronic Lower Limb Ischemia
Roman V. Deev et al.
JOURNAL OF CARDIOVASCULAR PHARMACOLOGY AND THERAPEUTICS (2015)
Talimogene Laherparepvec Improves Durable Response Rate in Patients With Advanced Melanoma
Robert H. I. Andtbacka et al.
JOURNAL OF CLINICAL ONCOLOGY (2015)
Genome Editing Technologies: Defining a Path to Clinic
Jacqueline Corrigan-Curay et al.
MOLECULAR THERAPY (2015)
Spinal Muscular Atrophy Therapeutics: Where do we Stand?
Constantin d'Ydewalle et al.
NEUROTHERAPEUTICS (2015)
Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study
Ole B. Suhr et al.
ORPHANET JOURNAL OF RARE DISEASES (2015)
TISSUEGENE-C (INVOSSA™) IN PATIENTS WITH OSTEOARTHRITIS: A PHASE II TRIALS
J. Cho et al.
OSTEOARTHRITIS AND CARTILAGE (2015)
Preliminary results of a phase II randomized study to determine the efficacy and safety of genetically engineered allogeneic human chondrocytes expressing TGF-β1 in patients with grade 3 chronic degenerative joint disease of the knee
J. J. Cherian et al.
OSTEOARTHRITIS AND CARTILAGE (2015)
Development and Application of an Ultrasensitive Hybridization-Based ELISA Method for the Determination of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides
Umar Burki et al.
NUCLEIC ACID THERAPEUTICS (2015)
Key points of basic theories and clinical practice in rAd-p53 (Gendicine™) gene therapy for solid malignant tumors
Yi Li et al.
EXPERT OPINION ON BIOLOGICAL THERAPY (2015)
Molecular Therapeutic Strategies for Spinal Muscular Atrophies: Current and Future Clinical Trials
Chiara Zanetta et al.
CLINICAL THERAPEUTICS (2014)
Spinal Muscular Atrophy: Therapeutic Strategies
Diana Castro et al.
CURRENT TREATMENT OPTIONS IN NEUROLOGY (2014)
Of CARs and TRUCKs: chimeric antigen receptor (CAR) T cells engineered with an inducible cytokine to modulate the tumor stroma
Markus Chmielewski et al.
IMMUNOLOGICAL REVIEWS (2014)
Spinal muscular atrophy: a motor neuron disorder or a multi-organ disease?
Monir Shababi et al.
JOURNAL OF ANATOMY (2014)
Clinical utility of recombinant adenoviral human p53 gene therapy: current perspectives
Guang-xia Chen et al.
ONCOTARGETS AND THERAPY (2014)
Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis
Wan Ling Wong et al.
LANCET GLOBAL HEALTH (2014)
Immune responses to AAV-vectors, the Glybera example from bencn to bedside
Valerie Ferreira et al.
FRONTIERS IN IMMUNOLOGY (2014)
Anti-vascular endothelial growth factor for neovascular age-related macular degeneration
Sharon D. Solomon et al.
COCHRANE DATABASE OF SYSTEMATIC REVIEWS (2014)
Primary analysis of a phase 1b multicenter trial to evaluate safety and efficacy of talimogene laherparepvec (T-VEC) and ipilimumab (ipi) in previously untreated, unresected stage IIIB-IV melanoma.
Igor Puzanov et al.
JOURNAL OF CLINICAL ONCOLOGY (2014)
Eteplirsen for the Treatment of Duchenne Muscular Dystrophy
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2013)
Clinical pharmacological properties of mipomersen (Kynamro), a second generation antisense inhibitor of apolipoprotein B
Stanley T. Crooke et al.
BRITISH JOURNAL OF CLINICAL PHARMACOLOGY (2013)
Diagnosis and treatment of familial hypercholesterolaemia
G. Kees Hovingh et al.
EUROPEAN HEART JOURNAL (2013)
Newborn bloodspot screening for Duchenne Muscular Dystrophy: 21 years experience in Wales (UK)
Stuart J. Moat et al.
EUROPEAN JOURNAL OF HUMAN GENETICS (2013)
Drug safety evaluation of defibrotide
Paul G. Richardson et al.
EXPERT OPINION ON DRUG SAFETY (2013)
Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial
D. Gaudet et al.
GENE THERAPY (2013)
Mipomersen, an Apolipoprotein B Synthesis Inhibitor, Reduces Atherogenic Lipoproteins in Patients With Severe Hypercholesterolemia at High Cardiovascular Risk
Gregory S. Thomas et al.
JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY (2013)
Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy
Jerry R. Mendell et al.
MUSCLE & NERVE (2013)
Lessons Learned from the Clinical Development and Market Authorization of Glybera
Laura M. Bryant et al.
HUMAN GENE THERAPY CLINICAL DEVELOPMENT (2013)
Defibrotide Interferes With Several Steps of the Coagulation-Inflammation Cycle and Exhibits Therapeutic Potential to Treat Severe Malaria
Ivo M. B. Francischetti et al.
ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY (2012)
Homozygous familial hypercholesterolemia: Current perspectives on diagnosis and treatment
Frederick J. Raal et al.
ATHEROSCLEROSIS (2012)
T-cell suicide gene therapy prompts thymic renewal in adults after hematopoietic stem cell transplantation
Luca Vago et al.
BLOOD (2012)
Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency
Amel Hassan et al.
BLOOD (2012)
Mipomersen: A Safe and Effective Antisense Therapy Adjunct to Statins in Patients With Hypercholesterolemia
Daniel N. Ricotta et al.
CARDIOLOGY IN REVIEW (2012)
Apolipoprotein B Synthesis Inhibition With Mipomersen in Heterozygous Familial Hypercholesterolemia
Evan A. Stein et al.
CIRCULATION (2012)
Gene therapy for lipoprotein lipase deficiency
Daniel Gaudet et al.
CURRENT OPINION IN LIPIDOLOGY (2012)
Defibrotide for prophylaxis of hepatic veno-occlusive disease in paediatric haemopoietic stem-cell transplantation: an open-label, phase 3, randomised controlled trial
Selim Corbacioglu et al.
LANCET (2012)
Stem cells in severe infantile spinal muscular atrophy
Eugenio Mercuri et al.
NEUROMUSCULAR DISORDERS (2012)
Randomized, Placebo-Controlled Trial of Mipomersen in Patients with Severe Hypercholesterolemia Receiving Maximally Tolerated Lipid-Lowering Therapy
Mary P. McGowan et al.
PLOS ONE (2012)
Familial Hypercholesterolemia: Screening, diagnosis and management of pediatric and adult patients Clinical guidance from the National Lipid Association Expert Panel on Familial Hypercholesterolemia
Anne C. Goldberg et al.
JOURNAL OF CLINICAL LIPIDOLOGY (2011)
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
Sebahattin Cirak et al.
LANCET (2011)
Local and Distant Immunity Induced by Intralesional Vaccination with an Oncolytic Herpes Virus Encoding GM-CSF in Patients with Stage IIIc and IV Melanoma
Howard L. Kaufman et al.
ANNALS OF SURGICAL ONCOLOGY (2010)
Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease and Multiorgan Failure after Stem Cell Transplantation: A Multicenter, Randomized, Dose-Finding Trial
Paul G. Richardson et al.
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2010)
Rexin-G, a targeted genetic medicine for cancer
Erlinda M. Gordon et al.
EXPERT OPINION ON BIOLOGICAL THERAPY (2010)
Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial
Frederick J. Raal et al.
LANCET (2010)
Advanced Phase I/II Studies of Targeted Gene Delivery In Vivo: Intravenous Rexin-G for Gemcitabine-resistant Metastatic Pancreatic Cancer
Sant P. Chawla et al.
MOLECULAR THERAPY (2010)
Cross-species comparison of in vivo PK/PD relationships for second-generation antisense oligonucleotides targeting apolipoprotein B-100
Rosie Z. Yu et al.
BIOCHEMICAL PHARMACOLOGY (2009)
Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA
Virginia B. Mattis et al.
BMC NEUROSCIENCE (2009)
Oral Administration of the Thyrotropin-Releasing Hormone (TRH) Analogue, Taltireline Hydrate, in Spinal Muscular Atrophy
Zenichiro Kato et al.
JOURNAL OF CHILD NEUROLOGY (2009)
Phase II Clinical Trial of a Granulocyte-Macrophage Colony-Stimulating Factor-Encoding, Second-Generation Oncolytic Herpesvirus in Patients With Unresectable Metastatic Melanoma
Neil N. Senzer et al.
JOURNAL OF CLINICAL ONCOLOGY (2009)
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. (vol 8, pg 918, 2009)
M. Kinali et al.
LANCET NEUROLOGY (2009)
Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I-II study
Fabio Ciceri et al.
LANCET ONCOLOGY (2009)
Ranibizumab versus Verteporfin Photodynamic Therapy for Neovascular Age-Related Macular Degeneration: Two-Year Results of the ANCHOR Study
David M. Brown et al.
OPHTHALMOLOGY (2009)
Introduction: Understanding the Role of Angiogenesis and Antiangiogenic Agents in Age-Related Macular Degeneration
Susan B. Bressler
OPHTHALMOLOGY (2009)
A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy
Natalia N. Singh et al.
RNA BIOLOGY (2009)
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
Maria Kinali et al.
LANCET NEUROLOGY (2009)
The natural history and prognosis of neovascular age-related macular degeneration
Tien Wong et al.
OPHTHALMOLOGY (2008)
Dystrophin, its interactions with other proteins, and implications for muscular dystrophy
James M. Ervasti
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2007)
Risk factors for acute leukemia in children: A review
Martin Belson et al.
ENVIRONMENTAL HEALTH PERSPECTIVES (2007)
A phase I study of OncoVEXGM-CSF, a second-generation oncolytic herpes simplex virus expressing granulocyte macrophage colony-stimulating factor
Jennifer C. C. Hu et al.
CLINICAL CANCER RESEARCH (2006)
Effects of Onyx-015 among metastatic colorectal cancer patients that have failed prior treatment with 5-FU/leucovorin
TR Reid et al.
CANCER GENE THERAPY (2005)
HIV-associated retinopathy in the HAART era
DE Goldberg et al.
RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES (2005)
An apolipoprotein B antisense oligonucleotide lowers LDL cholesterol in hyperlipidemic mice without causing hepatic steatosis
RM Crooke et al.
JOURNAL OF LIPID RESEARCH (2005)
Molecular and functional analysis of intragenic SMN1 mutations in patients with spinal muscular atrophy
Y Sun et al.
HUMAN MUTATION (2005)
Current status of gendicine in China: Recombinant human Ad-p53 agent for treatment of cancers
ZH Peng
HUMAN GENE THERAPY (2005)
Prevention of veno-occlusive disease with defibrotide after allogeneic stem cell transplantation
Y Chalandon et al.
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2004)
Pegaptanib for neovascular age-related macular degeneration
ES Gragoudas et al.
NEW ENGLAND JOURNAL OF MEDICINE (2004)
Intra-tumor injection of H101, a recombinant adenovirus, in combination with chemotherapy in patients with advanced cancers: A pilot phase II clinical trial
Wei Lu et al.
WORLD JOURNAL OF GASTROENTEROLOGY (2004)
Defibrotide in the treatment of children with veno-occlusive disease (VOD): a retrospective multicentre study demonstrates therapeutic efficacy upon early intervention
S Corbacioglu et al.
BONE MARROW TRANSPLANTATION (2004)
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy
L Brichta et al.
HUMAN MOLECULAR GENETICS (2003)
The effect of cytomegalovirus retinitis on the quality of life of patients with AIDS in the era of highly active antiretroviral therapy
JH Kempen et al.
OPHTHALMOLOGY (2003)
Increased vascularity detected by digital subtraction angiography after VEGF gene transfer to human lower limb artery:: A randomized, placebo-controlled, double-blinded phase II study
K Mäkinen et al.
MOLECULAR THERAPY (2002)
Treatment of spinal muscular atrophy by sodium butyrate
JG Chang et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2001)
Pharmacokinetics and safety of an anti-vascular endothelial growth factor aptamer (NX1838) following injection into the vitreous humor of rhesus monkeys
DW Drolet et al.
PHARMACEUTICAL RESEARCH (2000)
Defibrotide for the treatment of hepatic veno-occlusive disease: results of the European compassionate-use study
R Chopra et al.
BRITISH JOURNAL OF HAEMATOLOGY (2000)
Changes in the natural history of cytomegalovirus retinitis following the introduction of highly active antiretroviral therapy
JR Deayton et al.
AIDS (2000)
The incidence of cytomegalovirus (CMV) antigenemia and CMV disease is reduced by highly active antiretroviral therapy
S Varani et al.
EUROPEAN JOURNAL OF EPIDEMIOLOGY (2000)
Drug therapy: Age-related macular degeneration.
SL Fine et al.
NEW ENGLAND JOURNAL OF MEDICINE (2000)