Journal ArticleDOI
State-of-the-art gene-based therapies: the road ahead.
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TLDR
Improvements in the gene transfer vectors used in therapeutic trials have led to substantial clinical successes in patients with serious genetic conditions, such as immunodeficiency syndromes, blindness and some cancer types.Abstract:
Improvements in the gene transfer vectors used in therapeutic trials have led to substantial clinical successes in patients with serious genetic conditions, such as immunodeficiency syndromes, blindness and some cancer types. Several barriers need to be overcome before this type of therapy becomes a widely accepted treatment for a broad group of medical diseases. However, recent progress in the field is finally realizing some of the promises made more than 20 years ago, providing optimism for additional successes in the near future.read more
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Journal ArticleDOI
Non-coding RNAs in human disease
TL;DR: Dysregulation of these ncRNAs is being found to have relevance not only to tumorigenesis, but also to neurological, cardiovascular, developmental and other diseases, and there is great interest in therapeutic strategies to counteract these perturbations.
Journal ArticleDOI
Non-viral vectors for gene-based therapy
Hao Yin,Rosemary Lynn Kanasty,Ahmed A. Eltoukhy,Arturo J. Vegas,J. Robert Dorkin,Daniel G. Anderson +5 more
TL;DR: The biological barriers to gene delivery in vivo are introduced and recent advances in material sciences, nanotechnology and nucleic acid chemistry that have yielded promising non-viral delivery systems are discussed, some of which are currently undergoing testing in clinical trials.
In vivo genome editing using Staphylococcus aureus Cas9
F. Ann Ran,Le Cong,Winston X. Yan,David A. Scott,Jonathan S. Gootenberg,Andrea J. Kriz,Bernd Zetsche,Ophir Shalem,Xuebing Wu,Kira S. Makarova,Eugene V. Koonin,Phillip A. Sharp,Feng Zhang +12 more
TL;DR: In this paper, the RNA-guided endonuclease Cas9 has emerged as a versatile genome-editing platform and has been used for basic research and therapeutic applications that use the highly versatile adeno-associated virus (AAV) delivery vehicle.
Journal ArticleDOI
In vivo genome editing using Staphylococcus aureus Cas9
F. Ann Ran,Le Cong,Winston X. Yan,David A. Scott,Jonathan S. Gootenberg,Andrea J. Kriz,Bernd Zetsche,Ophir Shalem,Xuebing Wu,Kira S. Makarova,Eugene V. Koonin,Phillip A. Sharp,Feng Zhang +12 more
TL;DR: Six smaller Cas9 orthologues are characterized and it is shown that Cas9 from Staphylococcus aureus (SaCas9) can edit the genome with efficiencies similar to those of SpCas9, while being more than 1 kilobase shorter.
Journal ArticleDOI
Current advances and future perspectives in extrusion-based bioprinting.
TL;DR: This paper, presenting a first-time comprehensive review of EBB, discusses the current advancements in EBB technology and highlights future directions to transform the technology to generate viable end products for tissue engineering and regenerative medicine.
References
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Journal ArticleDOI
A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease.
Shin-ichi Muramatsu,Ken-ichi Fujimoto,Seiya Kato,Hiroaki Mizukami,Sayaka Asari,Kunihiko Ikeguchi,Tadataka Kawakami,Masashi Urabe,Akihiro Kume,Toshihiko Sato,Watanabe Eiju,Keiya Ozawa,Imaharu Nakano +12 more
TL;DR: The findings provide class IV evidence regarding the safety and efficacy of AADC gene therapy and warrant further evaluation in a randomized, controlled, phase 2 setting.
Book ChapterDOI
The Mechanism of Naked DNA Uptake and Expression
Jon A. Wolff,Vladimir G. Budker +1 more
TL;DR: The administration of naked nucleic acids into animals is increasingly being used as a research tool to elucidate mechanisms of gene expression and the role of genes and their cognate proteins in the pathogenesis of disease in animal models and is proving to be a very useful research tool.
Journal ArticleDOI
Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: Implications for gene therapy using AAV vectors.
Christine L. Halbert,A. Dusty Miller,Sharon McNamara,Julia Emerson,Ronald L. Gibson,Bonnie W. Ramsey,Moira L. Aitken +6 more
TL;DR: The results indicate that AAV type 2, 5 or 6 exposure is low in CF and control populations and even lower in CF children, as measured in serum from children and adults with CF and from normal adults.
Journal ArticleDOI
Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression
Zejing Wang,Christian S. Kuhr,Christian S. Kuhr,James M. Allen,Michael J. Blankinship,Paul Gregorevic,Jeffrey S. Chamberlain,Stephen J. Tapscott,Stephen J. Tapscott,Rainer Storb,Rainer Storb +10 more
TL;DR: It is demonstrated that a brief course of immunosuppression with a combination of anti-thymocyte globulin, cyclosporine, and mycophenolate mofetil is sufficient to permit long-term and robust expression of a canine micro-dystrophin (c-micro-dys) transgene in the skeletal muscle of a dog model for DMD.
Journal ArticleDOI
Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy
Annette Deichmann,Salima Hacein-Bey-Abina,Manfred Schmidt,Manfred Schmidt,Alexandrine Garrigue,Martijn H. Brugman,Jingqiong Hu,Hanno Glimm,Gabor Gyapay,Bernard Prum,Christopher Fraser,Nicolas Fischer,Kerstin Schwarzwaelder,Kerstin Schwarzwaelder,Maria Luise Siegler,Dick de Ridder,Dick de Ridder,Karin Pike-Overzet,Steven J. Howe,Adrian J. Thrasher,Gerard Wagemaker,Ulrich Abel,Frank J. T. Staal,Eric Delabesse,Jean-Luc Villeval,Bruce J. Aronow,Christophe Hue,Claudia Prinz,Manuela Wissler,Chuck Klanke,Jean Weissenbach,Ian E. Alexander,Alain Fischer,Christof von Kalle,Marina Cavazzana-Calvo +34 more
TL;DR: The results obtained from a large-scale mapping of retroviral integration sites isolated from cells of 9 patients with X-linked SCID (SCID-X1) treated with a retrovirus-based gene therapy protocol help to elucidate the relationship between vector insertion and long-term in vivo selection of transduced cells in human patients with SCID- X1.