Search Results - "Gene therapy"

Current status and perspective of CAR-T and CAR-NK cell therapy trials in Germany by Albinger, Nawid, Hartmann, Jessica, Ullrich, Evelyn

“…Chimeric antigen receptor (CAR)-T cell therapies are on the verge of becoming powerful immunotherapeutic tools for combating hematological diseases confronted…”
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Inside out: optimization of lipid nanoparticle formulations for exterior complexation and in vivo delivery of saRNA by Blakney, Anna K., McKay, Paul F., Yus, Bárbara Ibarzo, Aldon, Yoann, Shattock, Robin J.

“…Self-amplifying RNA (saRNA) is a promising biotherapeutic tool that has been used as a vaccine against both infectious diseases and cancer. saRNA has been…”
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Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems by Guan, S, Rosenecker, J

“…Because of its safe and effective protein expression profile, in vitro transcribed messenger RNA (IVT-mRNA) represents a promising candidate in the development…”
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CARAMBA: a first-in-human clinical trial with SLAMF7 CAR-T cells prepared by virus-free Sleeping Beauty gene transfer to treat multiple myeloma by Prommersberger, Sabrina, Reiser, Michael, Beckmann, Julia, Danhof, Sophia, Amberger, Maximilian, Quade-Lyssy, Patricia, Einsele, Hermann, Hudecek, Michael, Bonig, Halvard, Ivics, Zoltán

“…Clinical development of chimeric antigen receptor (CAR)-T-cell therapy has been enabled by advances in synthetic biology, genetic engineering, clinical-grade…”
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Prime editing – an update on the field by Scholefield, Janine, Harrison, Patrick T.

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Gene therapy and gene correction: targets, progress, and challenges for treating human diseases by Cring, Matthew R., Sheffield, Val C.

“…The field of gene therapy has made significant strides over the last several decades toward the treatment of previously untreatable genetic disease. Gene…”
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Understanding the molecular biology of intervertebral disc degeneration and potential gene therapy strategies for regeneration: a review by Sampara, Prasanthi, Banala, Rajkiran Reddy, Vemuri, Satish Kumar, AV, Gurava Reddy, GPV, Subbaiah

“…Intervertebral disc degeneration (IVDD) is a multi-factorial process characterized by phenotypic and genotypic changes, which leads to low back pain and…”
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How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy by Singh, N N, Howell, M D, Androphy, E J, Singh, R N

“…Spinal muscular atrophy (SMA), a prominent genetic disease of infant mortality, is caused by low levels of survival motor neuron (SMN) protein owing to…”
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Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters by Nieuwenhuis, Bart, Haenzi, Barbara, Hilton, Sam, Carnicer-Lombarte, Alejandro, Hobo, Barbara, Verhaagen, Joost, Fawcett, James W.

“…Adeno-associated viral vectors are widely used as vehicles for gene transfer to the nervous system. The promoter and viral vector serotype are two key factors…”
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Excision of HIV-1 DNA by gene editing: a proof-of-concept in vivo study by Kaminski, R, Bella, R, Yin, C, Otte, J, Ferrante, P, Gendelman, H E, Li, H, Booze, R, Gordon, J, Hu, W, Khalili, K

“…A CRISPR/Cas9 gene editing strategy has been remarkable in excising segments of integrated HIV-1 DNA sequences from the genome of latently infected human cell…”
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Harnessing the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated Cas9 system to disrupt the hepatitis B virus by Zhen, S, Hua, L, Liu, Y-H, Gao, L-C, Fu, J, Wan, D-Y, Dong, L-H, Song, H-F, Gao, X

“…The current therapies to treat hepatitis B virus (HBV) infection are limited. Recently, clustered regularly interspaced short palindromic repeat (CRISPR)…”
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Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial by Gaudet, D, Méthot, J, Déry, S, Brisson, D, Essiembre, C, Tremblay, G, Tremblay, K, de Wal, J, Twisk, J, van den Bulk, N, Sier-Ferreira, V, van Deventer, S

“…We describe the 2-year follow-up of an open-label trial (CT-AMT-011–01) of AAV1-LPL S447X gene therapy for lipoprotein lipase (LPL) deficiency (LPLD), an…”
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Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry by Wu, Di, Hwang, Philsang, Li, Tiansen, Piszczek, Grzegorz

“…Recombinant adeno-associated viruses (rAAV) are used extensively as gene delivery vectors in clinical studies, and several rAAV based treatments have already…”
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The clinical landscape for SMA in a new therapeutic era by Talbot, K, Tizzano, E F

“…Despite significant advances in basic research, the treatment of degenerative diseases of the nervous system remains one of the greatest challenges for…”
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Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates by Gray, S J, Nagabhushan Kalburgi, S, McCown, T J, Jude Samulski, R

“…Injection of adeno-associated virus (AAV) into the cerebrospinal fluid (CSF) offers a means to achieve widespread transgene delivery to the central nervous…”
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Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions by Riyad, Jalish M., Weber, Thomas

“…In the last two decades, recombinant adeno-associated virus has emerged as the most popular gene therapy vector. Recently AAV gene therapy has been approved by…”
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Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system by DeRosa, F, Guild, B, Karve, S, Smith, L, Love, K, Dorkin, J R, Kauffman, K J, Zhang, J, Yahalom, B, Anderson, D G, Heartlein, M W

“…DNA-based gene therapy has considerable therapeutic potential, but the challenges associated with delivery continue to limit progress. Messenger RNA (mRNA) has…”
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Lentivirus pre-packed with Cas9 protein for safer gene editing by Choi, J G, Dang, Y, Abraham, S, Ma, H, Zhang, J, Guo, H, Cai, Y, Mikkelsen, J G, Wu, H, Shankar, P, Manjunath, N

“…The CRISPR/Cas9 system provides an easy way to edit specific site/s in the genome and thus offers tremendous opportunity for human gene therapy for a wide…”
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MiR-21 derived from the exosomes of MSCs regulates the death and differentiation of neurons in patients with spinal cord injury by Kang, Jian, Li, Zhenhuan, Zhi, Zhongzheng, Wang, Shiqiang, Xu, Guanghui

“…In this study, we aimed to investigate the therapeutic effect of miR-21 in the treatment of spinal cord injury (SCI) as well as its underlying molecular…”
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Non-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system by O’Keeffe Ahern, Jonathan, Lara-Sáez, Irene, Zhou, Dezhong, Murillas, Rodolfo, Bonafont, Jose, Mencía, Ángeles, García, Marta, Manzanares, Darío, Lynch, Jennifer, Foley, Ruth, Xu, Qian, Sigen, A, Larcher, Fernando, Wang, Wenxin

“…Recent advances in molecular biology have led to the CRISPR revolution, but the lack of an efficient and safe delivery system into cells and tissues continues…”
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