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    • Molecular therapy. Methods & clinical development Journal

    Papers
    Efficient Enrichment of Gene-Modified Primary T Cells Via CCR5-Targeted Integration of Mutant Dihydrofolate Reductase
    Biswajit Paul,Guillermo S. Romano Ibarra,Nicholas Hubbard,Teresa Einhaus,Alexander Astrakhan,David J. Rawlings,Hans-Peter Kiem,Christopher W. Peterson
    Molecular Therapy — Methods & Clinical Development（2018）
    Targeted gene therapy strategies utilizing homology-driven repair (HDR) allow for greater control over transgene integration site, copy number, and expression-significant advantages over traditional vector-mediated gene therapy with random genome integration. However, the relativ...
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    Pre-TCRα Supports CD3-dependent Reactivation and Expansion of TCRα-deficient Primary Human T-cells
    Roman Galetto,Celine Lebuhotel,Laurent Poirot,Agnes Gouble,Maria L. Toribio,Julianne Smith,Andrew Scharenberg
    Molecular Therapy — Methods & Clinical Development（2014）
    Chimeric antigen receptor technology offers a highly effective means for increasing the anti-tumor effects of autologous adoptive T-cell immunotherapy, and could be made widely available if adapted to the use of allogeneic T-cells. Although gene-editing technology can be used to ...
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    Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with Β-Thalassemia Major
    Nikoletta Psatha,Andreas Reik,Susan Phelps,Yuanyue Zhou,Demetri Dalas,Evangelia Yannaki,Dana N. Levasseur,Fyodor D. Urnov,Michael C. Holmes,Thalia Papayannopoulou
    Molecular Therapy — Methods & Clinical Development（2018）
    In the present report, we carried out clinical-scale editing in adult mobilized CD34+ hematopoietic stem and progenitor cells (HSPCs) using zinc-finger nuclease-mediated disruption of BCL11 a to upregulate the expression of gamma-globin (fetal hemoglobin). In these cells, disrupt...
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    Toward a Rapid Production of Multivirus-Specific T Cells Targeting BKV, Adenovirus, CMV, and EBV from Umbilical Cord Blood
    Hema Dave,Min Luo,J. W. Blaney,Shabnum Patel,Cecilia Barese,Conrad Russell Cruz,Elizabeth J. Shpall,Catherine M. Bollard,Patrick J. Hanley
    Molecular Therapy — Methods & Clinical Development（2017）
    Umbilical cord blood (CB) has emerged as an effective alternative donor source for hematopoietic stem cell transplantation. Despite this success, the prolonged duration of immune suppression following CB transplantation and the naiveté of CB T cells leave patients susceptible to ...
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    A Chimeric Virus-Based Probe Unambiguously Detects Live Circulating Tumor Cells with High Specificity and Sensitivity
    Xinping Fu,Lihua Tao,Xiaoliu Zhang
    SSRN Electronic Journal（2021）
    The current methods for detecting circulating tumor cells (CTCs) suffer from several drawbacks. We report a novel method that is based on a chimeric virus probe and can detect CTCs with extremely high specificity and sensitivity. Moreover, it exclusively detects live CTCs, and it...
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    Novel Non-integrating DNA Nano-S/MAR Vectors Restore Gene Function in Isogenic Patient-Derived Pancreatic Tumor Models
    Matthias Bozza,Edward W. Green,Elisa Espinet,Alice De Roia,Corinna Klein,Vanessa Vogel,Rienk Offringa,James A. Williams,Martin Sprick,Richard P. Harbottle
    Molecular Therapy - Methods & Clinical Development（2020）
    We describe herein non-integrating minimally sized nano-S/MAR DNA vectors, which can be used to genetically modify dividing cells in place of integrating vectors. They represent a unique genetic tool, which avoids vector-mediated damage. Previous work has shown that DNA vectors c...
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    Development and Validation of qPCR and RT-qPCR: Regulatory Points to Consider When Conducting Biodistribution and Vector Shedding Studies of Gene and Cell Therapy Products
    Haiyan Ma,Kristin N. Bell,Rossi N. Loker
    Molecular therapy. Methods & clinical development（2020）
    Abstract Gene and cell therapy fields have experienced remarkable growth over the past decade. Demand for preclinical and clinical safety assessments of these cell and gene therapy test articles (TAs) have effectively increased the necessity for regulated biodistribution, vecto...
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    Prostaglandin E2 As Transduction Enhancer Affects Competitive Engraftment of Human Hematopoietic Stem and Progenitor Cells
    Valentina Poletti,Annita Montepeloso,Danilo Pellin,Alessandra Bif
    MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT（2023）
    Ex vivo gene therapy (GT) is a promising treatment for inherited genetic diseases. An ideal transduction protocol should determine high gene marking in long-term self-renewing hematopoietic stem cells (HSCs), preserving their repopulation potential during in vitro manipulation. I...
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    A C1qTNF3 Collagen Domain Fusion Chaperones Diverse Secreted Proteins and Anti-Aβ Scfvs: Applications for Gene Therapies
    Brenda D. Moore,Yong Ran,Marshall S. Goodwin, Kavitha Komatineni,Karen N. Mcfarland,Kristy Dillon, Caleb Charles,Danny Ryu,Xuefei Liu,Stefan Prokop,Benoit I. Giasson,Todd E. Golde,
    MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT（2023）
    Enhancing production of protein cargoes delivered by gene therapies can improve efficacy by reducing the amount of vector or simply increasing transgene expression levels. We explored the utility of a 126-amino acid collagen domain (CD) derived from the C1qTNF3 protein as a fusio...
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    Chronic Enzyme Replacement to the Brain of a Late Infantile Neuronal Ceroid Lipofuscinosis Mouse Has Differential Effects on Phenotypes of Disease
    Jennifer A. Wiseman,Yu Meng,Yuliya Nemtsova,Paul G. Matteson,James H. Millonig,Dirk F. Moore,David E. Sleat,Peter Lobel
    Molecular Therapy — Methods & Clinical Development（2017）
    Late infantile neuronal ceroid lipofuscinosis (LINCL) is a fatal inherited neurodegenerative disease caused by loss of lysosomal protease tripeptidyl peptidase 1 (TPP1). We have investigated the effects of chronic intrathecal (IT) administration using enzyme replacement therapy (...
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    Fbxw7-associated Drug Resistance is Reversed by Induction of Terminal Differentiation in Murine Intestinal Organoid Culture
    Federica Lorenzi,Roya Babaei-Jadidi,Jonathan Sheard,Bradley Spencer-Dene,Abdolrahman S. Nateri
    Molecular therapy Methods & clinical development（2016）
    Colorectal cancer (CRC) is one of the top three cancer-related causes of death worldwide. FBXW7 is a known tumor-suppressor gene, commonly mutated in CRC and in a variety of other epithelial tumors. Low expression of FBXW7 is also associated with poor prognosis. Loss of FBXW7 sen...
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    Engineering a Highly Durable Adeno-Associated Virus Receptor for Analytical Applications
    Kouhei Yoshida,Yuji Tsunekawa, Kento Kurihara, Kazuya Watanabe, Yuriko Makino-Manabe,Mikako Wada,Toru Tanaka,Teruhiko Ide,Takashi Okada
    Molecular Therapy — Methods & Clinical Development（2023）
    Adeno-associated virus (AAV) is a major viral vector used in gene therapy. There are multiple AAV serotypes, and many engineered AAV serotypes are developed to alter their tissue tropisms with capsid modification. The universal AAV receptor (AAVR) is an essential receptor for mul...
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    Current Landscape of Clinical Development and Approval of Advanced Therapies
    Carolina Iglesias-Lopez,Antonia Agusti,Antoni Vallano,Merce Obach
    Molecular Therapy Methods & Clinical Development（2021）
    Advanced therapy medicinal products (ATMPs) are innovative therapies that mainly target orphan diseases and high unmet medical needs. The uncertainty about the product's benefit-risk balance at the time of approval, the limitations of nonclinical development, and the complex qual...
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    Adeno-Associated Viral Vectors in Neuroscience Research
    David L. Haggerty,Gregory G. Grecco,Kaitlin C. Reeves,Brady Atwood
    Molecular Therapy - Methods & Clinical Development（2020）
    Adeno-associated viral vectors (AAVs) are increasingly useful preclinical tools in neuroscience research studies for interrogating cellular and neurocircuit functions and mapping brain connectivity. Clinically, AAVs are showing increasing promise as viable candidates for treating...
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    Transcutaneous Ultrasound-Mediated Nonviral Gene Delivery to the Liver in a Porcine Model
    Dominic M. Tran, Feng Zhang,Kyle P. Morrison,Keith R. Loeb,James Harrang,Masaki Kajimoto,Francisco Chavez,Li Wu,Carol H. Miao
    Molecular Therapy — Methods & Clinical Development（2019）
    Ultrasound (US)-mediated gene delivery (UMGD) of nonviral vectors was demonstrated in this study to be an effective method to transfer genes into the livers of large animals via a minimally invasive approach. We developed a transhepatic venous nonviral gene delivery protocol in c...
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    Atg5flox-Derived Autophagy-Deficient Model of Pompe Disease: Does It Tell the Whole Story
    Jeong-A Lim,Hossein Zare,Rosa Puertollano,Nina Raben
    Molecular therapy Methods & clinical development（2017）
    Defective autophagy is a prominent feature in Pompe disease, an inherited deficiency of acid alpha-glucosidase. An absence of this enzyme leaves cells unable to digest glycogen in the lysosome. The major tissues affected by glycogen accumulation are cardiac and skeletal muscles. ...
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    Membrane-stabilizing Copolymers Confer Marked Protection to Dystrophic Skeletal Muscle in Vivo
    Evelyne M. Houang,Karen J. Haman,Antonio Filareto,Rita C. Perlingeiro,Frank S. Bates,Dawn A. Lowe,Joseph M. Metzger
    The FASEB Journal（2015）
    Duchenne muscular dystrophy (DMD) is a fatal disease of striated muscle deterioration. A unique therapeutic approach for DMD is the use of synthetic membrane stabilizers to protect the fragile dystrophic sarcolemma against contraction-induced mechanical stress. Block copolymer-ba...
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    Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65
    Eloise Hudry,Eva Andres-Mateos,Eli P. Lerner,Adrienn Volak,Olivia Cohen,Bradley T. Hyman,Casey A. Maguire,Luk H. Vandenberghe
    Molecular therapy Methods & clinical development（2018）
    Adeno-associated viral vectors (AAVs) have demonstrated potential in applications for neurologic disorders, and the discovery that some AAVs can cross the blood-brain barrier (BBB) after intravenous injection has further expanded these opportunities for non-invasive brain deliver...
    Cited61Views0
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    Manufacturing of Recombinant Adeno-Associated Viral Vectors: New Technologies Are Welcome
    Eduard Ayuso
    Molecular Therapy — Methods & Clinical Development（2016）
    Recombinant adeno-associated viral vectors (rAAV) are probably the most powerful tools for in vivo gene delivery. Encouraging preclinical data have been followed by successful gene therapy clinical trials including Leber's congenital amaurosis type 2 (refs. 1Maguire AM High KA Au...
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    Tacrolimus-resistant SARS-CoV-2-specific T Cell Products to Prevent and Treat Severe COVID-19 in Immunosuppressed Patients
    L. Peter, D. Wendering,S. Schlickeiser,H. Hoffmann,R. Noster,D. Wagner,G. Zarrinrad, S. Muench,S. Schulenberg,M. -F. Mashreghi,P. Reinke,H. -D. Volk,
    Molecular therapy Methods & clinical development（2022）
    Solid organ transplant (SOT) recipients receive therapeutic immunosuppression that compromises their immune response to infections and vaccines. For this reason, SOT patients have a high risk of developing severe coronavirus disease 2019 (COVID-19) and an increased risk of death ...
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