Akil, Omar Blits, Bas Lustig, Lawrence R Leake, Patricia A
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Human gene therapy
Contemporary cochlear implants (CI) are generally very effective for remediation of severe to profound sensorineural hearing loss, but outcomes are still highly variable. Auditory nerve survival is likely one of the major factors underlying this variability. Neurotrophin therapy therefore has been proposed for CI recipients, with the goal of improv...
Giamouridis, Dimosthenis Gao, Mei Hua Lai, N Chin Tan, Zhen Kim, Young Chul Guo, Tracy Miyanohara, Atsushi Blankesteijn, Matthijs W Biessen, Erik A L Hammond, H Kirk
...
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Human gene therapy
Peptide infusions of peptides the corticotropin releasing factor family, including urocortin 2, stresscopin, and urocortin 3 (UCn3), have favorable acute effects in clinical heart failure (HF), but their short half-lives make them unsuitable for chronic therapy. This study asked whether UCn3 gene transfer, which provides sustained elevation of plas...
Perocheau, Dany P Cunningham, Sharon Lee, Juhee Antinao Diaz, Juan Waddington, Simon N Gilmour, Kimberly Eaglestone, Simon Lisowski, Leszek Thrasher, Adrian J Alexander, Ian E
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Human gene therapy
Recombinant adeno-associated virus (rAAV) vectors are a promising platform for in vivo gene therapy. The presence of neutralizing antibodies (Nab) against AAV capsids decreases cell transduction efficiency and is a common exclusion criterion for participation in clinical trials. Novel engineered capsids are being generated to improve gene delivery ...
VanLith, Caitlin Guthman, Rebekah Nicolas, Clara T Allen, Kari Du, Zeji Joo, Dong Jin Nyberg, Scott L Lillegard, Joseph B Hickey, Raymond D
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Human gene therapy
Hereditary tyrosinemia type 1 (HT1) is an autosomal recessive disorder caused by deficiency of fumarylacetoacetate hydrolase (FAH). It has been previously shown that ex vivo hepatocyte-directed gene therapy using an integrating lentiviral vector to replace the defective Fah gene can cure liver disease in small- and large-animal models of HT1. This ...
Venkatesan, Jagadeesh K Moutos, Franklin T Rey-Rico, Ana Estes, Bradley T Frisch, Janina Schmitt, Gertrud Madry, Henning Guilak, Farshid Cucchiarini, Magali
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Human gene therapy
Combining gene therapy approaches with tissue engineering procedures is an active area of translational research for the effective treatment of articular cartilage lesions, especially to target chondrogenic progenitor cells such as those derived from the bone marrow. This study evaluated the effect of genetically modifying concentrated human mesenc...
Liang, Xiao Liu, Li Wei, Yu-Quan Gao, Guang-Ping Wei, Xia-Wei
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Human gene therapy
Considerable efforts have been devoted to develop safe and efficient gene therapies for life-threatening or inherited diseases. The choice of gene delivery vehicle plays key roles in enhancing the therapeutic effect of nucleic acid cargo. To date, gene therapy approaches involving both viral vectors and nonviral vectors have been evaluated in clini...
Doering, Christopher B Denning, Gabriela Shields, Jordan E Fine, Eli J Parker, Ernest T Srivastava, Alok Lollar, Pete Spencer, H Trent
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Human gene therapy
Genetically modified, autologous hematopoietic stem and progenitor cells (HSPCs) represent a new class of genetic medicine. Following this therapeutic paradigm, we are developing a product candidate, designated CD68-ET3-LV CD34+, for the treatment of the severe bleeding disorder, hemophilia A. The product consists of autologous CD34+ cells transduc...
Liu, Shuying Wang, Shixia Lu, Shan
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Human Gene Therapy
In recent vaccine studies, DNA immunization was found to effectively stimulate both innate and adaptive immunities to elicit high levels of antigen-specific antibody responses. The DNA molecule itself can activate multiple pathways of innate immunity. The in vivo production of antigens allows for presentation by major histocompatibility complexes, ...
Cheng, Max A. Farmer, Emily Huang, Claire Lin, John Hung, Chien-Fu Wu, T.-C.
Published in
Human Gene Therapy
Human papillomavirus (HPV) has long been recognized as the causative agent of cervical cancer. High-risk HPV types 16 and 18 alone are responsible for over 70% of all cases of cervical cancers. More recently, HPV has been identified as an etiological factor for several other forms of cancers, including oropharyngeal, anogenital, and skin. Thus, the...
Elliott, Sarah T C Keaton, Amelia A Chu, Jacqueline D Reed, Charles C Garman, Bradley Patel, Ami Yan, Jian Broderick, Kate E Weiner, David B
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Human gene therapy
Influenza A H3N2 viruses circulate globally, leading to substantial morbidity and mortality. Commercially available, antigen-matched influenza vaccines must be updated frequently to match dynamic sequence variability in immune epitopes, especially within viral influenza A H3N2 hemagglutinin (H3). In an effort to create comprehensive immune response...
