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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Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways.
Dirk Grimm,Konrad L. Streetz,Konrad L. Streetz,Catherine L. Jopling,Theresa A. Storm,Kusum Pandey,Corrine R. Davis,Patricia L. Marion,Felix H. Salazar,Mark A. Kay +9 more
TL;DR: The risk of oversaturating endogenous small RNA pathways can be minimized by optimizing shRNA dose and sequence, as exemplified here by the report of persistent and therapeutic RNAi against human hepatitis B virus in vivo.
Journal ArticleDOI
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Nathalie Cartier,Salima Hacein-Bey-Abina,Cynthia C. Bartholomae,Gabor Istvan Veres,Manfred Schmidt,Ina Kutschera,Michel Vidaud,Ulrich Abel,Liliane Dal-Cortivo,Laure Caccavelli,Nizar Mahlaoui,Veronique Kiermer,Denice Mittelstaedt,Céline Bellesme,Najiba Lahlou,François Lefrère,Stéphane Blanche,Muriel Audit,Emmanuel Payen,Philippe Leboulch,Philippe Leboulch,Bruno l’Homme,Pierre Bougnères,Christof von Kalle,Alain Fischer,Marina Cavazzana-Calvo,Patrick Aubourg +26 more
TL;DR: Lentiviral-mediated gene therapy of hematopoietic stem cells can provide clinical benefits in ALD, and progressive cerebral demyelination in the two patients stopped, a clinical outcome comparable to that achieved by allogeneic HCT.
Journal ArticleDOI
Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen.
Laura A. Johnson,Richard A. Morgan,Mark E. Dudley,Lydie Cassard,James Chih-Hsin Yang,Michael S. Hughes,Udai S. Kammula,Richard E. Royal,Richard M. Sherry,John R. Wunderlich,Chyi-Chia Richard Lee,Nicholas P. Restifo,Susan L. Schwarz,Alexandria P. Cogdill,Rachel Bishop,Hung Kim,Carmen C. Brewer,Susan F. Rudy,Carter VanWaes,Jeremy L. Davis,Aarti Mathur,Robert T. Ripley,Debbie Ann N. Nathan,Carolyn M. Laurencot,Steven A. Rosenberg +24 more
TL;DR: T cells expressing highly reactive TCRs mediate cancer regression in humans and target rare cognate-antigen-containing cells throughout the body, a finding with important implications for the gene therapy of cancer.
Journal ArticleDOI
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Marina Cavazzana-Calvo,Emmanuel Payen,Olivier Negre,Gary P. Wang,Kathleen M. Hehir,Floriane Fusil,Julian D. Down,Maria Denaro,Troy Brady,Karen A. Westerman,Resy Cavallesco,Beatrix Gillet-Legrand,Laure Caccavelli,Laure Caccavelli,Riccardo Sgarra,Leila Maouche-Chretien,Françoise Bernaudin,Robert Girot,Ronald Dorazio,Geert Jan Mulder,Axel Polack,Arthur Bank,Jean Soulier,Jérôme Larghero,Nabil Kabbara,Bruno Dalle,Bernard Gourmel,Gérard Socié,Stany Chrétien,Stany Chrétien,Nathalie Cartier,Patrick Aubourg,Alain Fischer,Alain Fischer,Kenneth Cornetta,Frédéric Galactéros,Yves Beuzard,Eliane Gluckman,Frederick D. Bushman,Salima Hacein-Bey-Abina,Salima Hacein-Bey-Abina,Philippe Leboulch +41 more
TL;DR: It is shown that, 33 months after lentiviral β-globin gene transfer, an adult patient with severe βE/β0-thalassaemia dependent on monthly transfusions since early childhood has become transfusion independent for the past 21 months.
Journal ArticleDOI
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.
Marion Ott,Manfred Schmidt,Kerstin Schwarzwaelder,Stefan Stein,Ulrich Siler,Ulrike Koehl,Hanno Glimm,K. Kühlcke,Andrea Schilz,Hana Kunkel,Sonja Naundorf,Andrea Brinkmann,Annette Deichmann,Marlene Fischer,Claudia R. Ball,Ingo H. Pilz,Cynthia E. Dunbar,Yang Du,Nancy A. Jenkins,Neal G. Copeland,Ursula Lüthi,Moustapha Hassan,Adrian J. Thrasher,Dieter Hoelzer,Christof von Kalle,Reinhard Seger,Manuel Grez +26 more
TL;DR: The results suggest that gene therapy in combination with bone marrow conditioning can be successfully used to treat inherited diseases affecting the myeloid compartment such as CGD.