Gene therapy and molecular medicine

1995 ◽  
Vol 59 (S21A) ◽  
pp. 353-434
Keyword(s):  
Gene Therapy ◽  
Molecular Medicine

Molecular medicine. One less hoop for gene therapy

Science ◽  
1994 ◽  
Vol 265 (5172) ◽  
pp. 599-599 ◽  
Author(s):  
E Marshall
Keyword(s):  
Gene Therapy ◽  
Molecular Medicine

STATUS AND POTENTIAL OF GENE THERAPY IN CLINICAL MEDICINE

2002 ◽  
Vol 18 (3) ◽  
pp. 645-674 ◽  
Author(s):  
Anita Lyngstadaas
Keyword(s):  
Gene Therapy ◽  
Clinical Research ◽  
Clinical Medicine ◽  
Severe Combined Immunodeficiency ◽  
Molecular Medicine ◽  
Phase Iii ◽  
Somatic Therapy ◽  
Health Authorities ◽  
Three Phase ◽  
Somatic Gene

Objective: Somatic gene therapy is a new method in the rapidly expanding field of molecular medicine. Due to recent encouraging results and the promising prospect for some disease groups, Norwegian health authorities wanted to assess somatic therapy with evidence-based standards for strategic use. This article presents the results of this assessment, discussed in the context of the policy-making process in Norway, including ethical and legislational considerations.Methods: Clinical gene therapy protocols, ongoing or completed with published results, where available, were identified through a systematic survey of descriptive protocols and publications. Preclinical literature was also reviewed.Results: Gene therapy is dominated by preclinical and clinical research. Most of the gene therapy protocols identified are in early phases (phases I and II) with only a few patients in each study. Of the protocols included in the assessment, only three phase III studies are represented. Except for the use of soluble antisense oligonucleotides against cytomegalovirus eye infection, gene therapy is presently not an established treatment modality. Promising results have been observed in treatment of cancer and cardiovascular diseases and, most recently, in inherited severe combined immunodeficiency and hemophilia. Several interesting principles addressing a large panel of conditions are currently being developed and tested.Conclusions: Gene therapy is developing into an important medical concept that needs to be included within the Norwegian healthcare system. It is recommended that the Norwegian Ministry of Health and Social Affairs fund a national program to boost infrastructure in selected scientific groups both in preclinical and clinical research. The national procedures regulating approval of gene therapy trials should be made more efficient while at the same time allowing for proper control and ethical considerations. It is emphasized that gene therapy trials should be carefully monitored for side effects.

Prospects Of Non-Coding Elements In Genomic Dna Based Gene Therapy

2021 ◽  
Vol 21 ◽  
Author(s):  
Simna SP ◽  
Zongchao Han
Keyword(s):  
Gene Therapy ◽  
Genomic Dna ◽  
Insertional Mutagenesis ◽  
Molecular Medicine ◽  
Significant Development ◽  
Complementary Dna ◽  
A Genome ◽  
Next Generation Sequencing Ngs ◽  
Potential Use ◽  
Generation Sequencing

: Gene therapy has made significant development since the commencement of the first clinical trials a few decades ago and has remained a dynamic area of research regardless of obstacles such as immune response and insertional mutagenesis. Progression in various technologies like next-generation sequencing (NGS) and nanotechnology has established the importance of non-coding segments of a genome, thereby taking gene therapy to the next level. In this review, we have summarized the importance of non-coding elements, highlighting the advantages of using full-length genomic DNA loci (gDNA) compared to complementary DNA (cDNA) or minigene, currently used in gene therapy. The focus of this review is to provide an overview of the advances and the future of potential use of gDNA loci in gene therapy, expanding the therapeutic repertoire in molecular medicine.

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