Literature DB >> 24312844

Gene therapy, early promises, subsequent problems, and recent breakthroughs.

Saeideh Razi Soofiyani1, Behzad Baradaran, Farzaneh Lotfipour, Tohid Kazemi, Leila Mohammadnejad.   

Abstract

Gene therapy is one of the most attractive fields in medicine. The concept of gene delivery to tissues for clinical applications has been discussed around half a century, but scientist's ability to manipulate genetic material via recombinant DNA technology made this purpose to reality. Various approaches, such as viral and non-viral vectors and physical methods, have been developed to make gene delivery safer and more efficient. While gene therapy initially conceived as a way to treat life-threatening disorders (inborn errors, cancers) refractory to conventional treatment, to date gene therapy is considered for many non-life-threatening conditions including those adversely influence on a patient's quality of life. Gene therapy has made significant progress, including tangible success, although much slower than was initially predicted. Although, gene therapies still at a fairly primitive stage, it is firmly science based. There is justifiable hope that with enhanced pathobiological understanding and biotechnological improvements, gene therapy will be a standard part of clinical practice within 20 years.

Entities:  

Keywords:  Gene therapy; Non-viral vectors; Recombinant DNA technology; Viral vectors

Year:  2013        PMID: 24312844      PMCID: PMC3848228          DOI: 10.5681/apb.2013.041

Source DB:  PubMed          Journal:  Adv Pharm Bull        ISSN: 2228-5881


  63 in total

1.  LMO2 and gene therapy for severe combined immunodeficiency.

Authors:  Alain Fischer; Salima Hacein-Bey Abina; Adrian Thrasher; Christof von Kalle; Marina Cavazzana-Calvo
Journal:  N Engl J Med       Date:  2004-06-10       Impact factor: 91.245

Review 2.  Immune responses to gene therapy vectors: influence on vector function and effector mechanisms.

Authors:  N Bessis; F J GarciaCozar; M-C Boissier
Journal:  Gene Ther       Date:  2004-10       Impact factor: 5.250

Review 3.  Gene therapy of severe combined immunodeficiencies.

Authors:  M Cavazzana-Calvo; S Hacein-Bey; F Yates; J P de Villartay; F Le Deist; A Fischer
Journal:  J Gene Med       Date:  2001 May-Jun       Impact factor: 4.565

Review 4.  Gene therapy for cystic fibrosis: challenges and future directions.

Authors:  J M Wilson
Journal:  J Clin Invest       Date:  1995-12       Impact factor: 14.808

Review 5.  Prospects for gene therapy in cardiovascular disease.

Authors:  A Lafont; C Guérot; P Lemarchand
Journal:  Eur Heart J       Date:  1996-09       Impact factor: 29.983

6.  The kinetics of rAAV integration in the liver.

Authors:  C H Miao; R O Snyder; D B Schowalter; G A Patijn; B Donahue; B Winther; M A Kay
Journal:  Nat Genet       Date:  1998-05       Impact factor: 38.330

7.  Innate immune mechanisms dominate elimination of adenoviral vectors following in vivo administration.

Authors:  S Worgall; G Wolff; E Falck-Pedersen; R G Crystal
Journal:  Hum Gene Ther       Date:  1997-01-01       Impact factor: 5.695

8.  Gene therapy for cardiovascular disease.

Authors:  D W Muller
Journal:  Br Heart J       Date:  1994-10

Review 9.  Cancer gene therapy: an awkward adolescence.

Authors:  Michael M Gottesman
Journal:  Cancer Gene Ther       Date:  2003-07       Impact factor: 5.987

10.  Gene therapy for human genetic disease?

Authors:  T Friedmann; R Roblin
Journal:  Science       Date:  1972-03-03       Impact factor: 47.728

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  15 in total

1.  Using intron splicing trick for preferential gene expression in transduced cells: an approach for suicide gene therapy.

Authors:  F Pourzadegan; L Shariati; R Taghizadeh; H Khanahmad; Z Mohammadi; M A Tabatabaiefar
Journal:  Cancer Gene Ther       Date:  2015-12-18       Impact factor: 5.987

2.  Magnetofection Mediated Transient NANOG Overexpression Enhances Proliferation and Myogenic Differentiation of Human Hair Follicle Derived Mesenchymal Stem Cells.

Authors:  Seoyoung Son; Mao-Shih Liang; Pedro Lei; Xiaozheng Xue; Edward P Furlani; Stelios T Andreadis
Journal:  Bioconjug Chem       Date:  2015-03-10       Impact factor: 4.774

3.  Production and Use of Gesicles for Nucleic Acid Delivery.

Authors:  Mathias Mangion; Marc-André Robert; Igor Slivac; Rénald Gilbert; Bruno Gaillet
Journal:  Mol Biotechnol       Date:  2021-10-01       Impact factor: 2.695

4.  Obtaining a New Gene-Cell Construct Based on Transduced Olfactory Ensheathing Cells for the Treatment of Spinal Cord Injuries.

Authors:  A D Voronova; A O Sosnovtseva; O V Stepanova; A V Chadin; E K Karsuntseva; G A Fursa; I V Reshetov; V P Chekhonin
Journal:  Bull Exp Biol Med       Date:  2022-09-05       Impact factor: 0.737

Review 5.  Beyond GalNAc! Drug delivery systems comprising complex oligosaccharides for targeted use of nucleic acid therapeutics.

Authors:  Joseph O'Sullivan; Jose Muñoz-Muñoz; Graeme Turnbull; Neil Sim; Stuart Penny; Sterghios Moschos
Journal:  RSC Adv       Date:  2022-07-14       Impact factor: 4.036

6.  Protection is not always a good thing: The immune system's impact on gene therapy.

Authors:  Martiela Vaz de Freitas; Lariane Frâncio; Laura Haleva; Ursula da Silveira Matte
Journal:  Genet Mol Biol       Date:  2022-07-15       Impact factor: 2.087

Review 7.  Combination of the modulators of epigenetic machinery and specific cell signaling pathways as a promising approach for cell reprogramming.

Authors:  Arshak R Alexanian
Journal:  Mol Cell Biochem       Date:  2022-05-03       Impact factor: 3.842

8.  Lentiviral Transduction of Neuronal Cells.

Authors:  Anna Bellizzi; Nicholas Ahye; Hassen S Wollebo
Journal:  Methods Mol Biol       Date:  2021

Review 9.  Solid Lipid Nanoparticles as Efficient Drug and Gene Delivery Systems: Recent Breakthroughs.

Authors:  Jafar Ezzati Nazhad Dolatabadi; Hadi Valizadeh; Hamed Hamishehkar
Journal:  Adv Pharm Bull       Date:  2015-06-15

10.  Synthesis, characterization, and evaluation of poly (D,L-lactide-co-glycolide)-based nanoformulation of miRNA-150: potential implications for pancreatic cancer therapy.

Authors:  Sumit Arora; Suresh K Swaminathan; Ameya Kirtane; Sanjeev K Srivastava; Arun Bhardwaj; Seema Singh; Jayanth Panyam; Ajay P Singh
Journal:  Int J Nanomedicine       Date:  2014-06-18
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