scholarly journals Recent advances in cardiac gene therapy strategies targeting advanced heart failure

2017 ◽  
Vol 3 (4) ◽  
pp. 163-169 ◽  
Author(s):  
C. Chen ◽  
T. Seeger ◽  
V. Termglinchan ◽  
I. Karakikes
Keyword(s):  
Heart Failure ◽  
Gene Therapy ◽  
Advanced Heart Failure ◽  
Recent Advances ◽  
Cardiac Gene ◽  
Therapy Strategies

scholarly journals Gene therapy strategies for idiopathic pulmonary fibrosis: recent advances, current challenges, and future directions

2021 ◽  
Vol 20 ◽  
pp. 483-496
Author(s):  
Mitchel J.R. Ruigrok ◽  
Henderik W. Frijlink ◽  
Barbro N. Melgert ◽  
Peter Olinga ◽  
Wouter L.J. Hinrichs
Keyword(s):  
Gene Therapy ◽  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Future Directions ◽  
Recent Advances ◽  
Therapy Strategies

Potential HIV gene therapy strategies

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Cord Blood ◽  
Gene Editing ◽  
Mortality Risks ◽  
Recent Advances ◽  
Whole Exome ◽  
Localization Signal ◽  
Cord Blood Cells ◽  
The Cost ◽  
Therapy Strategies

The CCR5 null genotype generation has been a main focus in the HIV gene therapy industry. The presence of the X4 tropic virus, mobilization of HSPCs, the quality of the cells for manipulation, and gene editing efficiency appear to be the main obstacles in translating this technique. Unintended off-target cleavage is a key problem in CRISPR/Cas9 editing. With the development of small molecule expansion methods for cord blood HSPC, it would be advantageous to modify CCR5 in cord blood cells and expand them for transplantation. The generation of engraftable HSPCS from iPSCs would be an ideal technique for HSCC gene therapy.The haplotype-characterized iPSC would be the donor for many patients, and it could be a commercially available product. The 32 C CR5 homozygous people had no elevated mortality risks according to whole-exome sequencing and whole-genome genotyping, according to CCR 5 positive people, and had no higher mortality risks compared to those who were HIV positive. Recent advances in gene editing, such as non-viral delivery of Cas9 ribonucleoproteins, incorporation of a 3X-nuclear localization signal into spCas9, and use of HiFi Cas9 with chemically modified sgRNAs, can be combined with recent advances in transplantation. Infusing modest doses of gene modified primitive HSPC fractions indicated by CD34 + CD90 + CD45RA-, which can engraft better, is another option for lowering the cost of gene therapy.

scholarly journals Cardiac gene therapy of heart failure with phosphodiesterase PDE4B in mice

2019 ◽  
Vol 11 (2) ◽  
pp. 224-225
Author(s):  
A. Bourcier ◽  
C. Coquard ◽  
J. Margaria ◽  
S. Gomez ◽  
A. Varin ◽  
...  
Keyword(s):  
Heart Failure ◽  
Gene Therapy ◽  
Cardiac Gene

A novel paradigm for therapeutic basis of advanced heart failure—assessment by gene therapy

2005 ◽  
Vol 107 (1) ◽  
pp. 31-43 ◽  
Author(s):  
Tomie Kawada ◽  
Fujiko Masui ◽  
Hiroyuki Kumagai ◽  
Miki Koshimizu ◽  
Mikio Nakazawa ◽  
...  
Keyword(s):  
Heart Failure ◽  
Gene Therapy ◽  
Advanced Heart Failure ◽  
Failure Assessment

Cardiac gene therapy: Recent advances and future directions

2015 ◽  
Vol 215 ◽  
pp. 101-111 ◽  
Author(s):  
Daniel Mason ◽  
Yu-Zhe Chen ◽  
Harini Venkata Krishnan ◽  
Shilpa Sant
Keyword(s):  
Gene Therapy ◽  
Future Directions ◽  
Recent Advances ◽  
Cardiac Gene
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