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
Citations
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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
Progress towards liver and lung-directed gene therapy with helper-dependent adenoviral vectors.
Nicola Brunetti-Pierri,Philip Ng +1 more
TL;DR: Progress towards liver and lung directed gene therapy with HDAd as well as the current obstacles facing human applications and possible strategies to overcome these obstacles are discussed.
Journal ArticleDOI
Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B
Timothy C. Nichols,Robin A. Raymer,Helen W. G. Franck,Elizabeth P. Merricks,Dwight A. Bellinger,Natalie DeFriess,Paris Margaritis,Valder R. Arruda,Mark A. Kay,Katherine A. High,Katherine A. High +10 more
TL;DR: Although complete correction of coagulopathy has not been achieved, published data show that prophylactic factor replacement therapy and gene transfer can markedly reduce the frequency of spontaneous bleeding in haemophilic dogs.
Journal ArticleDOI
Minireview: Switching on progesterone receptor expression with duplex RNA.
TL;DR: Evidence now supports the existence of regulatory pathways that use small duplex RNAs to control transcription, and focuses on the robust and potent agRNA-mediated regulation of progesterone receptor.
Journal ArticleDOI
Gene therapy for recessive dystrophic epidermolysis bullosa.
TL;DR: The different gene therapy approaches that have been used for the treatment of dystrophic epidermolysis bullosa and the new perspectives that they open are discussed.
Book ChapterDOI
Fiber-modified adenoviruses for targeted gene therapy.
Hongju Wu,David T. Curiel +1 more
TL;DR: This chapter will focus on the genetic fiber modification strategy and provide a detailed protocol for generation of fiber-modified Ad5 vectors.