26 papers · ranked by Valyu relevance
Giulliana Augusta Rangel Gonçalves, Raquel de Melo Alves Paiva
The ability to make site-specific modifications to the human genome has been an objective in medicine since the recognition of the gene as the basic unit of heredity. Thus, gene therapy is understood as the ability of genetic improvement through the correction of altered (mutated) genes or site-specific modifications…
Tingting Wu, Yu Hu, Liang V. Tang
Gene therapy utilizes nucleic acid drugs to treat diseases, encompassing gene supplementation, gene replacement, gene silencing, and gene editing. It represents a distinct therapeutic approach from traditional medications and introduces novel strategies for genetic disorders. Over the past two decades, significant…
Lucía Bañuls, Daniel Pellicer, Silvia Castillo, María Mercedes Navarro-García + 3 more
'María Mercedes Navarro-García' 'María Magallón' 'Cruz González' 'Francisco Dasí'] Gene therapy is an alternative therapy in many respiratory diseases with genetic origin and currently without curative treatment. After five decades of progress, many different vectors and gene editing tools for genetic engineering are…
P. Saraswat, R. R. Soni, A. Bhandari, B. P. Nagori
Human gene therapy is the introduction of new genetic material into the cells of an individual with the intention of producing a therapeutic benefit for the patient. Deoxyribonucleic acid and ribonucleic acid are used in gene therapy. Over time and with proper oversight, human gene therapy might become an effective…
Nida Siddique, Hira Raza, Sehrish Ahmed, Zohaib Khurshid + 2 more
'Muhammad Sohail Zafar' 'Michael Barry'] Gene therapy holds a promising future for bridging the gap between the disciplines of medicine and clinical dentistry. The dynamic treatment approaches of gene therapy have been advancing by leaps and bounds. They are transforming the conventional approaches into more precise…
Praveen Halagali, Raagul Seenivasan, Amatha Sreedevi, Virendra S. Ligade + 4 more
Gene and cell therapies have emerged as transformative approaches for treating a wide range of genetic and acquired diseases. Central to their success is the development of safe and effective gene delivery systems, categorized broadly into viral and non-viral vectors. Each system has its own advantages and limitations…
Marta Carvalho, Bruno Sepodes, Ana Paula Martins
Advanced therapy medicinal products (ATMPs) have a massive potential to address existing unmet medical needs. Specifically, gene therapy medicinal products (GTMPs) may potentially provide cure for several genetic diseases. In Europe, the ATMP regulation was fully implemented in 2009 and, at this point, the Committee…
Wei-Kai Hua, Jeff C. Hsu, Yi-Chun Chen, Peter S. Chang + 6 more
Recent advances in gene therapy have brought novel treatment options for cancer. However, the full potential of this approach has yet to be unlocked due to the safety concerns and limited payload capacity of commonly utilized viral vectors. Virus-free DNA transposons, including piggyBac, have potential to obviate these…
Authors not listed
In this report, we examine the extensive research landscape of CRISPR with an emphasis on CRISPR therapeutics and showcase our results from an in-depth analysis of the most up-to-date scientific information consisting of more than 53,000 publications encompassing academic journal articles and patents, spanning nearly…
Bo Li, Ning Gao, Zhuang Zhang, Qian-Ming Chen + 3 more
'Yi Li' 'Michael Barry'] Based on the theoretical and clinical development of modern medicines, gene therapy has been a promising treatment strategy for cancer and other diseases. The practice of gene therapy is nearly 27 years old, since the first authorized gene transfer study took place at the National Institute of…
Rohini Prakash, Roderick Slavcev
Gene therapy offers immense potential for treating various diseases, including cancer, immunodeficiencies, and cardiovascular conditions. The efficacy of gene therapy (GT) largely depends on the vector used for gene delivery. Viral vectors, while effective, pose risks including insertional mutagenesis, immune…
Jonas Kolibius, Fabian Weiss, Patrick C. Freitag, Andreas Plückthun
High-capacity adenoviral (HC-AdV) vectors offer large transgene capacities and long-term expression of therapeutics, but require high doses due to limited transgene expression. In contrast, replication-competent AdV (RC-AdV) vectors enhance in situ transgene expression by genome replication and increased transcription…
Mohammed Aledhari, Mohamed Rahouti
Future Directions Authors: ['Mohammed Aledhari' 'Mohamed Rahouti'] Abstract—Gene and RNA editing methods, technologies, and applications are emerging as innovative forms of therapy and medicine, offering more efficient implementation compared to traditional pharmaceutical treatments. Current trends emphasize the urgent…
Nikoletta Psatha, Pavel Sova, Grigorios Georgolopoulos, Kiriaki Paschoudi + 12 more
Regulation of gene expression during cell development and differentiation is chiefly orchestrated by distal noncoding regulatory elements that precisely modulate cell selective gene activity. Gene therapy vectors rely on the cellular and context specificity of regulatory DNA elements to express therapeutic transgenes…
Jicong Cao, Eva Maria Novoa, Zhizhuo Zhang, William C.W. Chen + 6 more
Despite significant clinical progress in cell and gene therapies, maximizing protein expression in order to enhance potency remains a major challenge. One approach to increase protein expression is by optimizing translation through the engineering of 5’ untranslated regions (5’ UTRs). Here, we developed a…
Khishigjargal Batjargal, Tomoki Togashi, Yuji Kashiwakura, Nemekhbayar Baatartsogt + 8 more
Gene knock-in therapy has the potential to cure inherited liver diseases but is limited by low efficiency and delivery complexity. Here, we developed a single adeno-associated virus (AAV) vector system comprising a compact CRISPR effector, enAsCas12f, a guide RNA, and a donor template to enable therapeutic genome…
Liming Luo, Jocelyn Duen-Ya Jea, Yan Wang, Pei-Wen Chao + 1 more
The ability to control the expression of a therapeutic gene or a transgene in mammalian cells is crucial for safe and efficacious gene and cell therapy, as well as for elucidating the function of a specific gene product. Yet current mammalian gene regulation systems either evoke harmful immune responses in hosts or…
Timothy C. Stutz, Janet S. Sinsheimer, Mary E. Sehl, Jason Xu
Multitype branching processes are ideal for studying the population dynamics of stem cell populations undergoing mutation accumulation over the years following transplant. In such stochastic models, several quantities are of clinical interest as insertional mutagenesis carries the potential threat of leukemogenesis…
Nasir Uddin, Md Abu Monsur Dinar, Daniel Pack, Jason DeRouchey
Polyethylenimine (PEI), a widely used cationic polymer for nonviral gene delivery, often undergoes modification to enhance its transfection efficiency while reducing cytotoxicity. This study systematically examines the effects of acetylation and succinylation on PEI’s physicochemical properties, transfection…
Sai Guna Ranjan Gurazada, H Kennedy, Richard D. Braatz, Steven J. Mehrman + 2 more
adeno-associated virus (rAAV) gene therapy manufacturing Authors: ['Sai Guna Ranjan Gurazada' 'H Kennedy' 'Richard D. Braatz' 'Steven J. Mehrman' 'Shawn W. Polson' 'Irene Rombel'] Gene therapy is poised to transition from niche to mainstream medicine, with recombinant adeno-associated virus (rAAV) as the vector of…
Harrison Lawson, Huy Nguyen, Ayesha Tupe, Mariah Arral + 4 more
Over the last two decades, the utility of metal-organic frameworks (MOFs) has expanded from catalysis and gas storage to biomedical applications such as drug delivery. At first, MOF-based delivery platforms focused on delivering small molecules, with current work focusing on nucleic acids, such as DNA, short guide RNA…
Janet Sasso, Barbara Ambrose, Rumiana Tenchov, Ruchira Datta + 3 more
In the last decade, there has been a shift in research, clinical development, and commercial activity to exploit the many roles of RNA in physiology for use in medicine. With the rapid success in the development of lipid-RNA nanoparticles for mRNA vaccines against COVID-19 and with several approved RNA-based drugs, RNA…
Tianyu Pan, Xiang Zhang, Weining Shen, Ting Ye
Application to a study of Long-Term Outcomes in a Hemophilia Gene Therapy Trial Authors: ['Tianyu Pan' 'Xiang Zhang' 'Weining Shen' 'Ting Ye'] Gene therapies aim to address the root causes of diseases, particularly those stemming from rare genetic defects that can be life-threatening or severely debilitating. While…
Josh Smith, Sara Carillo, Aditya Kulkarni, Erin Redman + 2 more
Adeno-associated viruses (AAVs) are viral vectors used as delivery systems for gene therapies. Intact protein characterization of AAV viral capsid proteins (VPs) and their post-translational modifications is critical to ensuring product quality. In this study, microchip based ZipChip capillary electrophoresis-mass…
Manish Kumar
Cancer is a complex disease having a number of composite problems to be considered including cancer immune evasion, therapy resistance, and recurrence for a cure. Fundamentally, it remains a genetic disease as diverse aspects of the complexity of tumor growth and cancer development relate to its genetic machinery and…
Soren Ghorai, Ashley Pearson
Chimeric antigen receptor T (CAR-T) cell therapy has transformed the field of immunology by redirecting T lymphocytes toward tumor antigens. Despite successes in attaining remission rates as high as 70%, the performance of CAR therapy is limited by the survival of T cells. T cell persistence is crucial as it sustains…