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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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
Gene therapy finds its niche
TL;DR: Gene therapy is finally poised to make a contribution to the treatment of debilitating, highly penetrant genetic diseases that have proved intractable to other regimens.
Journal ArticleDOI
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
Mark L. Brantly,Jeffrey D. Chulay,Lili Wang,Christian Mueller,Margaret Humphries,L. Terry Spencer,Farshid N. Rouhani,Thomas J. Conlon,Roberto Calcedo,Michael R. Betts,Carolyn T. Spencer,Barry J. Byrne,James M. Wilson,Terence R. Flotte +13 more
TL;DR: It is suggested that immune responses to AAV capsid that develop after i.m. injection of a serotype 1 rAAV vector expressing AAT do not completely eliminate transduced cells in this context.
Journal ArticleDOI
A robust system for production of minicircle DNA vectors
TL;DR: A genetically modified Escherichia coli strain is constructed that stably expresses a set of inducible minicircle-assembly enzymes, ΦC31 integrase and I-SceI homing endonuclease, making it feasible to use minicircles in place of plasmids in mammalian transgene expression studies.
Journal ArticleDOI
DNA electrotransfer: its principles and an updated review of its therapeutic applications.
Franck M. Andre,Lluis M. Mir +1 more
TL;DR: The evolution in the concepts and the various optimization steps that have led to the use of combinations of pulses that fit with the known roles of the electric pulses in DNA electrotransfer, namely cell electropermeabilization and DNA electrophoresis are described.
Journal ArticleDOI
CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression.
Stephen C. Hyde,Ian A. Pringle,Syahril Abdullah,Syahril Abdullah,Anna E. Lawton,Lee A. Davies,Anusha Varathalingam,Graciela A Nunez-Alonso,Anne-Marie Green,Reto P. Bazzani,Stephanie G Sumner-Jones,Mario Chan,Hongyu Li,Nelson S. Yew,Seng H. Cheng,A. Christopher Boyd,Jane C. Davies,Uta Griesenbach,David J. Porteous,David N. Sheppard,Felix M. Munkonge,Eric W.F.W. Alton,Deborah R. Gill +22 more
TL;DR: Using a CpG-free pDNA expression vector, sustained in vivo transgene expression is achieved in the absence of lung inflammation and it is demonstrated that retention of even a single CPG in pDNA is sufficient to elicit an inflammatory response, whereas C pG- free pDNA vectors do not.