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Published in 2019 at "Biotechnology Letters"
DOI: 10.1007/s10529-019-02732-z
Abstract: As more and more cell and gene therapies are being developed and with the increasing number of regulatory approvals being obtained, there is an emerging and pressing need for industrial translation. Process efficiency, associated cost…
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Keywords:
gene;
cell gene;
gene therapy;
therapy manufacturing ... See more keywords
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Published in 2020 at "Cytotherapy"
DOI: 10.1016/j.jcyt.2020.03.204
Abstract: Background & Aim Lack of comprehensive product knowledge in clinical applications may result in toxicity or reduced drug efficacy. The distributive fate and retention of cell/gene therapies is rarely evaluated, with very few studies assessing…
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Keywords:
biodistribution;
product;
cell gene;
bioprocess solutions ... See more keywords
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Published in 2020 at "Cytotherapy"
DOI: 10.1016/j.jcyt.2020.03.351
Abstract: Background & Aim Cell and Gene Therapies are currently being developed to treat different diseases. By design, those therapies have very short shelf lives compared to traditional sterile pharmaceutical drugs. Besides, Cell and Gene Therapies…
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Keywords:
sterility test;
sterility;
cell;
cell gene ... See more keywords
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Published in 2020 at "Value in Health"
DOI: 10.1016/j.jval.2020.08.1839
Abstract: Objectives: To explore the long-term impact of Covid-19 on the pricing, market access, and payer evidence requirements in rare diseases and cell/gene-therapies Methods: A survey-based Covid-19 payer impact tracker covering US and EU (n= 80…
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Keywords:
impact;
gene therapies;
cell gene;
pricing market ... See more keywords
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Published in 2020 at "Nature Communications"
DOI: 10.1038/s41467-020-20096-1
Abstract: Cell and gene therapies offer opportunities for treating disease with potential to restore function, and cure disease. However, they are not without risk and pose complex logistical, economic, ethical and social challenges for health systems.…
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Keywords:
patient public;
systematic review;
cell gene;
gene ... See more keywords
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Published in 2020 at "Human gene therapy"
DOI: 10.1089/hum.2020.236
Abstract: Cell and gene therapy (CGT) has a variety of potential applications in human immunodeficiency virus (HIV) prevention and management. The existing network of CGT researchers for HIV cure will benefit from an influx of fresh…
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Keywords:
hiv cure;
gene;
cell gene;
engaging cell ... See more keywords
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Published in 2022 at "Human gene therapy"
DOI: 10.1089/hum.2022.210
Abstract: : Solid cancers remain a major health challenge in terms of research, not only due to their structure and organisation but also in the molecular and genetic variations present between tumours as well as within…
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Keywords:
gene biotherapies;
pancreatic cancer;
cell gene;
cancer cell ... See more keywords
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Published in 2021 at "Briefings in bioinformatics"
DOI: 10.1093/bib/bbab295
Abstract: Single-cell RNA sequencing (scRNA-seq) technologies facilitate the characterization of transcriptomic landscapes in diverse species, tissues, and cell types with unprecedented molecular resolution. In order to evaluate various biological hypotheses using high-dimensional single-cell gene expression data,…
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Keywords:
single cell;
expression data;
cell gene;
gene expression ... See more keywords
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Published in 2022 at "Stem Cells Translational Medicine"
DOI: 10.1093/stcltm/szac046
Abstract: Abstract The development of cell-, gene- and tissue engineering (CGT)-based therapies must adhere to strict pharmaceutical quality management standards, as for any other biological or small-molecule drug. However, early developments often failed to fully comply…
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Keywords:
compliance non;
development;
development cell;
cell gene ... See more keywords
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Published in 2019 at "Cold Spring Harbor perspectives in biology"
DOI: 10.1101/cshperspect.a035667
Abstract: To date, more than 200 monogenic, often devastating, skin diseases have been described. Because of unmet medical needs, development of long-lasting and curative therapies has been consistently attempted, with the aim of correcting the underlying…
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Keywords:
cell gene;
gene therapy;
combined cell;
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Published in 2021 at "IEEE/ACM transactions on computational biology and bioinformatics"
DOI: 10.1109/tcbb.2021.3098394
Abstract: Clustering single-cell RNA sequence (scRNA-seq) data poses statistical and computational challenges due to their high-dimensionality and data-sparsity, also known as 'dropout' events. Recently, Regularized Auto-Encoder (RAE) based deep neural network models have achieved remarkable success…
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Keywords:
clustering single;
cell;
single cell;
cell gene ... See more keywords