Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

iPS Cell Differentiation01:22

iPS Cell Differentiation

2.2K
The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
2.2K
EPS and iPS Cells in Disease Research01:21

EPS and iPS Cells in Disease Research

3.2K
Embryonic and induced pluripotent stem cells are excellent models for disease research because of their ability to self-renew and differentiate into most cell types. Somatic cells from a patient are isolated and reprogrammed into induced pluripotent stem cells or iPSCs. These iPSCs are later differentiated into the desired cell type, which mirrors the diseased cell of the patient. In this way, disease models have been created for investigating diseases such as Down syndrome, type I diabetes,...
3.2K
Stem Cell Culture01:17

Stem Cell Culture

4.5K
Stem cell research aims to find ways to use stem cells to regenerate and repair cellular damage. Over time, most adult cells undergo the wear and tear of aging and lose their ability to divide and repair themselves. Stem cells do not display a particular morphology or function. Adult stem cells, which exist as a small subset of cells in most tissues, keep dividing and can differentiate into a number of specialized cells generally formed by that tissue. These cells enable the body to renew and...
4.5K
Induced Pluripotent Stem Cells01:06

Induced Pluripotent Stem Cells

4.8K
Stem cells are undifferentiated cells that divide and produce different cell types. Ordinarily, cells that have differentiated into a specific cell type are terminally differentiated; however, scientists have found a way to reprogram these mature cells so that they dedifferentiate and return to an unspecialized, proliferative state. These cells are pluripotent like embryonic stem cells—able to produce all cell types—and are called induced pluripotent stem cells (iPSCs).
Somatic...
4.8K
Induced Pluripotent Stem Cells01:13

Induced Pluripotent Stem Cells

22.9K
Stem cells are undifferentiated cells that divide and produce different types of cells. Ordinarily, cells that have differentiated into a specific cell type are post-mitotic—that is, they no longer divide. However, scientists have found a way to reprogram these mature cells so that they “de-differentiate” and return to an unspecialized, proliferative state. These cells are also pluripotent like embryonic stem cells—able to produce all cell types—and are therefore...
22.9K
Embryonic Stem Cells00:58

Embryonic Stem Cells

25.8K
Embryonic stem (ES) cells are undifferentiated pluripotent cells, meaning they can produce any cell type in the body. This gives them tremendous potential in science and medicine since they can generate specific cell types for use in research or to replace body cells lost due to damage or disease.
25.8K

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Treatment of spinal muscolar atrophy with intrathecal mesenchymal cells.

International journal of stem cells·2013
Same author

The immunosuppressive effect of Wharton's jelly stromal cells depends on the timing of their licensing and on lymphocyte activation.

Cytotherapy·2010
Same author

Medium-term survival without haematopoietic stem cell transplantation in a case of IPEX: insights into nutritional and immunosuppressive therapy.

European journal of pediatrics·2007
Same author

Long-lasting CD3+ T-cell deficiency after cord blood stem cell transplantation in a human herpesvirus 6-infected child.

Journal of clinical microbiology·2005
Same author

Hemorrhagic cystitis in children undergoing bone marrow transplantation: a putative role for simian virus 40.

Transplantation·2004
Same author

Failure of a sibling umbilical cord blood transplantation to correct hemophilia A.

Haematologica·2004

Related Experiment Video

Updated: Apr 28, 2026

Generation of Induced Neural Stem Cells from Peripheral Mononuclear Cells and Differentiation Toward Dopaminergic Neuron Precursors for Transplantation Studies
12:13

Generation of Induced Neural Stem Cells from Peripheral Mononuclear Cells and Differentiation Toward Dopaminergic Neuron Precursors for Transplantation Studies

Published on: July 11, 2019

6.6K

Stem cells and niemann pick disease.

Marino Andolina1

  • 1Stamina Foundation, Italy.

International Journal of Stem Cells
|June 13, 2014
PubMed
Summary

Mesenchymal cell therapy shows promise for Niemann-Pick A disease, improving patient outcomes after bone marrow transplant failure. This cellular therapy offers a potential new treatment option for this rare genetic disorder.

Area of Science:

  • Biomedical research
  • Cellular therapy
  • Rare genetic disorders

Background:

  • Niemann-Pick A disease (NPA) is a fatal neurodegenerative disorder.
  • Characterized by progressive sphingomyelin accumulation in organs.
  • Typically limits patient survival to three years.

Purpose of the Study:

  • To describe the outcome of a patient with Niemann-Pick A disease.
  • To evaluate the efficacy of mesenchymal cell therapy following bone marrow transplantation.

Main Methods:

  • Patient underwent haploidentical bone marrow transplantation.
  • Subsequently received intrathecal and intravenous mesenchymal cell injections.

Main Results:

  • Bone marrow transplantation was unsuccessful.
Keywords:
MesenchymalNiemann PickStem cellsTreatment

More Related Videos

An Enzyme- and Serum-free Neural Stem Cell Culture Model for EMT Investigation Suited for Drug Discovery
07:43

An Enzyme- and Serum-free Neural Stem Cell Culture Model for EMT Investigation Suited for Drug Discovery

Published on: August 23, 2016

8.8K
GM-Free Generation of Blood-Derived Neuronal Cells
08:11

GM-Free Generation of Blood-Derived Neuronal Cells

Published on: February 13, 2021

2.5K

Related Experiment Videos

Last Updated: Apr 28, 2026

Generation of Induced Neural Stem Cells from Peripheral Mononuclear Cells and Differentiation Toward Dopaminergic Neuron Precursors for Transplantation Studies
12:13

Generation of Induced Neural Stem Cells from Peripheral Mononuclear Cells and Differentiation Toward Dopaminergic Neuron Precursors for Transplantation Studies

Published on: July 11, 2019

6.6K
An Enzyme- and Serum-free Neural Stem Cell Culture Model for EMT Investigation Suited for Drug Discovery
07:43

An Enzyme- and Serum-free Neural Stem Cell Culture Model for EMT Investigation Suited for Drug Discovery

Published on: August 23, 2016

8.8K
GM-Free Generation of Blood-Derived Neuronal Cells
08:11

GM-Free Generation of Blood-Derived Neuronal Cells

Published on: February 13, 2021

2.5K
  • Mesenchymal cell therapy led to psychomotor and storage improvements within one month.
  • Resolved hypersplenism, increasing platelet count from 20,000 to 120,000/microliter.

    • Conclusions:

    • Mesenchymal cell therapy demonstrated positive therapeutic effects in NPA.
    • Cellular therapy presents a potential treatment strategy for Niemann-Pick A disease.