Venkatesan, Jagadeesh Kumar Cai, Xiaoyu Meng, Weikun Rey-Rico, Ana Schmitt, Gertrud Speicher-Mentges, Susanne Falentin-Daudré, Céline Leroux, Amélie Madry, Henning Migonney, Véronique
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Human gene therapy
Scaffold-guided viral gene therapy is a novel, powerful tool to enhance the processes of tissue repair in articular cartilage lesions via the delivery and overexpression of therapeutic genes in a non-invasive, controlled release manner based on a procedure that may protect the gene vehicles from undesirable host immune responses. In the present stu...
Selvaraj, Nagarathinam Wang, Chao-Kuei Bowser, Brian Broadt, Trevor L Shaban, Samir Burns, Jenna Saptharishi, Nirmala Pechan, Peter Golebiowski, Diane Alimardanov, Asaf
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Published in
Human gene therapy
Recombinant adeno-associated viral (rAAV) vector-based gene therapy has been adapted for use in more than 100 clinical trials. This is mainly because of its excellent safety profile, ability to target a wide range of tissues, stable transgene expression, and significant clinical benefit. However, the major challenge is to produce a high titer, high...
Potter, Rachael A Griffin, Danielle A Heller, Kristin N Peterson, Ellyn L Clark, Emma K Mendell, Jerry R Rodino-Klapac, Louise R
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Human gene therapy
Duchenne muscular dystrophy (DMD) is a rare, X-linked, fatal, degenerative neuromuscular disease caused by mutations in the DMD gene. More than 2,000 mutations of the DMD gene are responsible for progressive loss of muscle strength, loss of ambulation, and generally respiratory and cardiac failure by age 30. Recently, gene transfer therapy has rece...
Griffin, Danielle A Pozsgai, Eric R Heller, Kristin N Potter, Rachael A Peterson, Ellyn L Rodino-Klapac, Louise R
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Human gene therapy
Limb-girdle muscular dystrophy type 2D (LGMD2D) is a progressive muscular dystrophy that manifests with muscle weakness, respiratory abnormalities, and in rare cases cardiomyopathy. LGMD2D is caused by mutations in the SGCA gene resulting in loss of protein and concomitant loss of some or all components of the dystrophin-associated glycoprotein com...
McGrath, Kyle Dotti, Gianpietro
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Human gene therapy
Chimeric antigen receptor (CAR) T cell therapy has revolutionized the treatment of hematological malignancies, but solid tumors continue to pose significant challenges. Oncolytic viruses (OVs) have generated significant excitement in the field of cancer treatment recently. In particular, OVs can help CAR T cells overcome some of the immunosuppressi...
Cornu, Tatjana I Mussolino, Claudio Müller, Matthias C Wehr, Claudia Kern, Winfried V Cathomen, Toni
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Human gene therapy
Progress in antiretroviral therapy has considerably reduced mortality and notably improved the quality of life of individuals infected with human immunodeficiency virus (HIV) since the pandemic began some 40 years ago. However, drug resistance, treatment-associated toxicity, adherence to medication, and the need for lifelong therapy have remained m...
Sheikh Hosseini, Motahareh Larijani, Bagher Gholipoor Kakroodi, Zahra Shokoohi, Mahshid Moarefzadeh, Mohammad Sayahpour, Forough Azam Goodarzi, Parisa Arjmand, Babak
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Human gene therapy
Breast cancer is a heterogeneous disease which is the consequence of several genetic and environmental factors. Also, it is one of the most common causes of cancer death and second-leading cancer among women all around the world. Therefore, it is necessary to develop novel therapeutic approaches useful for the successful treatment of breast cancer....
Choi, Stephanie Pegues, Melissa A Lam, Norris Geldres, Claudia Vanasse, Danielle Kochenderfer, James N
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Human gene therapy
Chimeric antigen receptors are artificial fusion proteins that incorporate antigen-recognition domains and T-cell signaling domains. CD30 is a cell-surface protein expressed on Hodgkin lymphoma (HL), some T-cell lymphomas, and some B-cell lymphomas. CD30 has a restricted expression pattern in normal cells, so CD30 has good potential as a clinical t...
Cai, Jing Yan, Guangmei
Published in
Human gene therapy
Oncolytic virotherapy represents an ideal therapeutic platform for cancer in which natural or engineered viruses selectively replicate in and destroy tumor cells while sparing normal cells. Oncolytic virotherapy is considered as a key contributor in modern immunotherapy. However, several challenges remain with regard to exploiting the potential of ...
Piguet, Françoise de Saint Denis, Timothée Audouard, Emilie Beccaria, Kevin Arthur, André Wurtz, Guillaume Schatz, Raphael Alves, Sandro Sevin, Caroline Zerah, Michel
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Published in
Human gene therapy
For more than ten years, gene therapy for neurological diseases has experienced intensive research growth and more recently therapeutic interventions for multiple indicationsBeneficial results in several phase 1/2 clinical studies, together with improved vector technology have advanced gene therapy for the central nervous system (CNS) in a new era ...
