Cell Biology and Translational Medicine, Volume 14
Springer International Publishing
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| Chapter title |
Stem Cell Applications in Lysosomal Storage Disorders: Progress and Ongoing Challenges.
|
|---|---|
| Book title |
Cell Biology and Translational Medicine, Volume 14
|
| Published in |
Advances in experimental medicine and biology, May 2021
|
| DOI | 10.1007/5584_2021_639 |
| Pubmed ID | |
| Book ISBNs |
978-3-03-080491-6, 978-3-03-080492-3
|
| Authors |
Köse, Sevil, Aerts-Kaya, Fatima, Uçkan Çetinkaya, Duygu, Korkusuz, Petek, Sevil Köse, Fatima Aerts-Kaya, Duygu Uçkan Çetinkaya, Petek Korkusuz |
| Abstract |
Lysosomal storage disorders (LSDs) are rare inborn errors of metabolism caused by defects in lysosomal function. These diseases are characterized by accumulation of completely or partially degraded substrates in the lysosomes leading to cellular dysfunction of the affected cells. Currently, enzyme replacement therapies (ERTs), treatments directed at substrate reduction (SRT), and hematopoietic stem cell (HSC) transplantation are the only treatment options for LSDs, and the effects of these treatments depend strongly on the type of LSD and the time of initiation of treatment. However, some of the LSDs still lack a durable and curative treatment. Therefore, a variety of novel treatments for LSD patients has been developed in the past few years. However, despite significant progress, the efficacy of some of these treatments remains limited because these therapies are often initiated after irreversible organ damage has occurred.Here, we provide an overview of the known effects of LSDs on stem cell function, as well as a synopsis of available stem cell-based cell and gene therapies that have been/are being developed for the treatment of LSDs. We discuss the advantages and disadvantages of use of hematopoietic stem cell (HSC), mesenchymal stem cell (MSC), and induced pluripotent stem cell (iPSC)-related (gene) therapies. An overview of current research data indicates that when stem cell and/or gene therapy applications are used in combination with existing therapies such as ERT, SRT, and chaperone therapies, promising results can be achieved, showing that these treatments may result in alleviation of existing symptoms and/or prevention of progression of the disease. All together, these studies offer some insight in LSD stem cell biology and provide a hopeful perspective for the use of stem cells. Further development and improvement of these stem cell (gene) combination therapies may greatly improve the current treatment options and outcomes of patients with a LSD. |
X Demographics
The data shown below were collected from the profiles of 3 X users who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United States | 1 | 33% |
| Netherlands | 1 | 33% |
| Unknown | 1 | 33% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 2 | 67% |
| Scientists | 1 | 33% |
Mendeley readers
The data shown below were compiled from readership statistics for 6 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Unknown | 6 | 100% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Master | 2 | 33% |
| Researcher | 1 | 17% |
| Student > Bachelor | 1 | 17% |
| Unknown | 2 | 33% |
| Readers by discipline | Count | As % |
|---|---|---|
| Nursing and Health Professions | 2 | 33% |
| Neuroscience | 2 | 33% |
| Chemistry | 1 | 17% |
| Unknown | 1 | 17% |
Attention Score in Context
This research output has an Altmetric Attention Score of 1. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 29 June 2021.
All research outputs
#15,683,389
of 23,305,591 outputs
Outputs from Advances in experimental medicine and biology
#2,543
of 4,989 outputs
Outputs of similar age
#259,358
of 440,864 outputs
Outputs of similar age from Advances in experimental medicine and biology
#52
of 151 outputs
Altmetric has tracked 23,305,591 research outputs across all sources so far. This one is in the 22nd percentile – i.e., 22% of other outputs scored the same or lower than it.
So far Altmetric has tracked 4,989 research outputs from this source. They typically receive a little more attention than average, with a mean Attention Score of 6.2. This one is in the 37th percentile – i.e., 37% of its peers scored the same or lower than it.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 440,864 tracked outputs that were published within six weeks on either side of this one in any source. This one is in the 31st percentile – i.e., 31% of its contemporaries scored the same or lower than it.
We're also able to compare this research output to 151 others from the same source and published within six weeks on either side of this one. This one is in the 49th percentile – i.e., 49% of its contemporaries scored the same or lower than it.