Molecular Therapy

A comprehensive study of a 29-capsid AAV library in a non-human primate central nervous system.  29:2806-2820. 2021

Inhibition of lncRNA MAAT Controls Multiple Types of Muscle Atrophy by cis- and trans-Regulatory Actions.  29:1102-1119. 2021

A “Shot in the Arm” for Sickle Cell Disease.  29:416-417. 2021

Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy.  29:442-463. 2021

Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease..  29:691-701. 2021

Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV.  28:1753-1755. 2020

Generation and Profiling of Tumor-Homing Induced Neural Stem Cells from the Skin of Cancer Patients.  28:1614-1627. 2020

A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna.  28:411-421. 2020

miR-639 Expression Is Silenced by DNMT3A-Mediated Hypermethylation and Functions as a Tumor Suppressor in Liver Cancer Cells.  28:587-598. 2020

The SINE Compound KPT-350 Blocks Dystrophic Pathologies in DMD Zebrafish and Mice.  28:189-201. 2020

AKT3 Gene Transfer Promotes Anabolic Reprogramming and Photoreceptor Neuroprotection in a Pre-clinical Model of Retinitis Pigmentosa.  27:1313-1326. 2019

A Neutralizing Aptamer to TGFBR2 and miR-145 Antagonism Rescue Cigarette Smoke- and TGF-β-Mediated CFTR Expression.  27:442-455. 2019

Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness.  26:1581-1593. 2018

Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.  25:1866-1880. 2017

Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward.  25:1076-1094. 2017

Systemic Injection of RPE65-Programmed Bone Marrow-Derived Cells Prevents Progression of Chronic Retinal Degeneration.  25:917-927. 2017

HIV-specific Immunity Derived from Chimeric Antigen Receptor-engineered Stem Cells.  23:1358-1367. 2015

Complement inhibition prevents oncolytic vaccinia virus neutralization in immune humans and cynomolgus macaques.  23:1066-1076. 2015

VIP-expressing dendritic cells protect against spontaneous autoimmune peripheral polyneuropathy..  22:1353-1363. 2014

A phase 1 trial of oncolytic HSV-1, g207, given in combination with radiation for recurrent GBM demonstrates safety and radiographic responses.  22:1048-1055. 2014

Cytotoxic and regulatory properties of circulating Vδ1+ γδ t cells: A new player on the cell therapy field?.  22:1416-1422. 2014

Overcoming the cystic fibrosis sputum barrier to leading adeno-associated virus gene therapy vectors.  22:1484-1493. 2014

Phase 1 clinical trial of intratumoral reovirus infusion for the treatment of recurrent malignant gliomas in adults.  22:1056-1062. 2014

VIP-expressing dendritic cells protect against spontaneous autoimmune peripheral polyneuropathy.  22:1353-1363. 2014

Grape exosome-like nanoparticles induce intestinal stem cells and protect mice from DSS-induced colitis.  21:1345-1357. 2013

Inflammation, Apoptosis, and Necrosis Induced by Neoadjuvant Fas Ligand Gene Therapy Improves Survival of Dogs With Spontaneous Bone Cancer..  20:2234-2243. 2012

Corrigendum: Treatment of brain inflammatory diseases by delivering exosome encapsulated anti-inflammatory drugs from the nasal region to the brain.  20:239. 2012

Glucose regulated protein 78 diminishes α-synuclein neurotoxicity in a rat model of parkinson disease.  20:1327-1337. 2012

Inflammation, apoptosis, and necrosis induced by neoadjuvant fas ligand gene therapy improves survival of dogs with spontaneous bone cancer.  20:2234-2243. 2012

Switching on the lights: The use of optogenetics to advance retinal gene therapy.  19:1190-1192. 2011

Delayed dominant-negative TNF gene therapy halts progressive loss of nigral dopaminergic neurons in a rat model of parkinson's disease.  19:46-52. 2011

Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa.  19:642-649. 2011

Treatment of brain inflammatory diseases by delivering exosome encapsulated anti-inflammatory drugs from the nasal region to the brain.  19:1769-1779. 2011

Vaccine-induced T cells provide partial protection against high-dose rectal SIVmac239 challenge of rhesus macaques.  19:417-426. 2011

Enhanced Induction of HIV-specific Cytotoxic T Lymphocytes by Dendritic Cell-targeted Delivery of SOCS-1 siRNA..  18:2028-2037. 2010

Enhanced induction of HIV-specific cytotoxic t lymphocytes by dendritic cell-targeted delivery of SOCS-1 siRNA.  18:2028-2037. 2010

In Vivo RNAi-Mediated α-Synuclein Silencing Induces Nigrostriatal Degeneration..  18:1450-1457. 2010

Mesenchymal stem cells expressing osteogenic and angiogenic factors synergistically enhance bone formation in a mouse model of segmental bone defect.  18:1026-1034. 2010

Gene therapy for leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.  18:643-650. 2010

A novel nanoparticle drug delivery system: The anti-inflammatory activity of curcumin is enhanced when encapsulated in exosomes.  18:1606-1614. 2010

In vivo RNAi-mediated α-synuclein silencing induces nigrostriatal degeneration.  18:1450-1457. 2010

α-synuclein expression in rat substantia nigra suppresses phospholipase D2 toxicity and nigral neurodegeneration.  18:1758-1768. 2010

Erratum: "Regulation of adult hematopoietic stem cells fate for enhanced tissue-specific repair" (Molecular Theraphy (2009) 10.1038/mt.2009.145).  17:1832. 2009

Herpes simplex virus oncolytic therapy for pediatric malignancies.  17:1125-1135. 2009

Cell-based therapy for heart disease: A clinically oriented perspective.  17:758-766. 2009

Nigrostriatal rAAV-mediated GDNF overexpression induces robust weight loss in a rat model of age-related obesity.  17:980-991. 2009

Does the human skeletal muscle harbor the murine equivalents of cardiac precursor cells?.  17:733-741. 2009

Can mesenchymal stem cells induce tolerance to cotransplanted human embryonic stem cells?.  17:176-182. 2009

Correction of ADAMTS13 deficiency by in utero gene transfer of lentiviral vector encoding ADAMTS13 genes.  17:34-41. 2009

Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates.  17:327-333. 2009

Phase Ib trial of mutant herpes simplex virus G207 inoculated pre-and post-tumor resection for recurrent GBM.  17:199-207. 2009

Regulation of adult hematopoietic stem cells fate for enhanced tissue-specific repair.  17:1594-1604. 2009

Enhancement of cytotoxic T-lymphocyte response in aged mice by a novel treatment with recombinant AdIL-12 and wild-type adenovirus in rapid succession.  16:1500-1506. 2008

A phase I trial of intratumoral administration of reovirus in patients with histologically confirmed recurrent malignant gliomas.  16:627-632. 2008

Challenging the cardiac differentiation of bone marrow cells: A clinical perspective.  16:1000-1001. 2008

Engineering human tumor-specific cytotoxic T cells to function in a hypoxic environment.  16:599-606. 2008

Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer.  16:458-465. 2008

Systemic osteoprotegerin gene therapy restores tumor-induced bone loss in a therapeutic model of beast cancer bone metastasis.  16:871-878. 2008

Gene transfer to ocular stem cells by early gestational intraamniotic injection of lentiviral vector.  15:579-587. 2007

Oncolytic adenoviruses kill breast cancer initiating CD44⁺ CD24^-/low cells.  15:2088-2093. 2007

In Vivo Delivery of Recombinant Viruses to the Fetal Murine Cochlea: Transduction Characteristics and Long-Term Effects on Auditory Function.  14:328-335. 2006

Triple-Targeted Oncolytic Adenoviruses Featuring the Cox2 Promoter, E1A Transcomplementation, and Serotype Chimerism for Enhanced Selectivity for Ovarian Cancer Cells.  14:164-174. 2006

Safety of Recombinant Adeno-Associated Virus Type 2-RPE65 Vector Delivered by Ocular Subretinal Injection.  13:1074-1084. 2006

Determination of specific CD4 and CD8 T cell epitopes after AAV2- and AAV8-hF.IX gene therapy.  13:260-269. 2006

Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.  12:1072-1082. 2005

Genetic analysis of the antibody response to AAV2 and factor IX.  11:866-874. 2005

Intratumoral injection of INGN 241, a nonreplicating adenovector expressing the melanoma-differentiation associated gene-7 (mda-7/IL24): Biologic outcome in advanced cancer patients.  11:160-172. 2005

Adenovirus-mediated FLT1-targeted proapoptotic gene therapy of human prostate cancer.  10:1059-1070. 2004

A phase I open-label, dose-escalation, multi-institutional trial of injection with an E1B-attenuated adenovirus, ONYX-015, into the peritumoral region of recurrent malignant gliomas, in the adjuvant setting.  10:958-966. 2004

Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degeneration.  10:855-861. 2004

Effects of CFTR, interleukin-10, and Pseudomonas aeruginosa on gene expression profiles in a CF bronchial epithelial cell Line.  10:562-573. 2004

Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system.  10:302-317. 2004

Adaptation of baculovirus system for large-scale production of rAAV vector serotypes.  9:S161-S161. 2004

A novel ex vivo angiogenesis assay based on electroporation-mediated delivery of naked plasmid DNA to skeletal muscle.  9:464-474. 2004

In utero gene therapy rescues vision in a murine model of congenital blindness.  9:182-188. 2004

A canine conditionally replicating adenovirus for evaluating oncolytic virotherapy in a syngeneic animal model.  7:163-173. 2003

Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents.  6:490-494. 2002

Adenoviral gene therapy for renal cancer requires retargeting to alternative cellular receptors.  62:4273-4281. 2002

Pharmacological regulation of protein expression from adeno-associated viral vectors in the eye.  6:238-242. 2002

A deviant immune response to viral proteins and transgene product is generated on subretinal administration of adenovirus and adeno-associated virus.  5:125-132. 2002

Gene transfer to ovarian cancer versus normal tissues with fiber-modified adenoviruses.  5:695-704. 2002

Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vector.  5:154-160. 2002

AAV-mediated delivery of ciliary neurotrophic factor prolongs photoreceptor survival in the rhodopsin knockout mouse.  3:241-248. 2001

An adenovirus with enhanced infectivity mediates molecular chemotherapy of ovarian cancer cells and allows imaging of gene expression.  4:223-231. 2001

Characterization of the cyclooxygenase-2 promoter in an adenoviral vector and its application for the mitigation of toxicity in suicide gene therapy of gastrointestinal cancers.  3:385-394. 2001

Long-term protection of retinal structure but not function using rAAV.CNTF in animal models of retinitis pigmentosa.  4:461-472. 2001

A targetable, injectable adenoviral vector for selective gene delivery to pulmonary endothelium in vivo.  2:562-578. 2000

Adenoviral-mediated suicide gene therapy for ovarian cancer.  2:524-530. 2000

An adenovirus encoding proapoptotic Bax induces apoptosis and enhances the radiation effect in human ovarian cancer.  1:545-554. 2000

Development of a novel trans-lentiviral vector that affords predictable safety.  2:47-55. 2000

Gene therapy for ocular disease.  1:501-505. 2000