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

Stem Cell Therapy for Tissue Regeneration01:21

Stem Cell Therapy for Tissue Regeneration

4.7K
Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
4.7K
Factors Affecting α-Alkylation of Ketones: Choice of Base01:10

Factors Affecting α-Alkylation of Ketones: Choice of Base

3.6K
α-Alkylation of ketones is achieved in the presence of alkyl halides and a base. The reaction proceeds via the formation of an enolate ion followed by nucleophilic substitution. The choice of base employed is essential as it is the key factor in determining the reaction outcome.
The reaction involving bases like EtO− whose conjugate acid EtOH (pKa = 15.9) is stronger than the ketone (pKa = 19.2) results in an equilibrium mixture with higher ketone concentration. As a consequence,...
3.6K
Adult Stem Cells01:33

Adult Stem Cells

33.9K
Stem cells are undifferentiated cells that divide and produce more stem cells or progenitor cells that differentiate into mature, specialized cell types. All the cells in the body are generated from stem cells in the early embryo, but small populations of stem cells are also present in many adult tissues including the bone marrow, brain, skin, and gut. These adult stem cells typically produce the various cell types found in that tissue—to replace cells that are damaged or to continuously...
33.9K
Base-Promoted α-Halogenation of Aldehydes and Ketones00:51

Base-Promoted α-Halogenation of Aldehydes and Ketones

4.2K
α-Halogenation of aldehydes and ketones is a reaction involving the substitution of α hydrogens with halogens in the presence of a base.  The reaction begins with the abstraction of  α hydrogen by the base to produce a nucleophilic enolate ion. This intermediate undergoes a subsequent nucleophilic substitution with the halogen to produce a monohalogenated carbonyl compound. If the starting substrate has more than one α hydrogen, it is difficult to stop the reaction...
4.2K
Embryonic Stem Cells00:58

Embryonic Stem Cells

32.7K
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.
32.7K
Embryonic Stem Cells00:57

Embryonic Stem Cells

5.3K
Embryonic stem (ES) cells were first discovered in mice in 1981 by Martin Evans. In 1998, James Thomson identified a method to isolate embryonic stem cells from humans. Human embryonic stem cells (hESCs) are obtained from 3-5 day old embryos that remain unused after an in vitro fertilization procedure.
ES cells are grown in a culture medium where they can divide indefinitely, creating ES cell lines. Under certain conditions, ES cells can differentiate, either spontaneously into a variety of...
5.3K

You might also read

Related Articles

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

Sort by
Same author

Meta-Analysis: Redefining Liver Disease Risk in Heterozygous Alpha-1 Antitrypsin Deficiency.

Alimentary pharmacology & therapeutics·2026
Same author

BMN 349, a small molecule inhibitor of Z alpha-1 antitrypsin polymerization, increases secretion and reduces intrahepatic inclusions in a mouse model of disease.

Hepatology (Baltimore, Md.)·2026
Same author

Z variant heterozygosity in alpha-1 antitrypsin deficiency: disease risk and treatment implications.

Orphanet journal of rare diseases·2026
Same author

Prevalence of liver disease and liver transplantation in pediatric ZZ alpha-1 antitrypsin deficiency: A systematic review and meta-analysis.

Digestive and liver disease : official journal of the Italian Society of Gastroenterology and the Italian Association for the Study of the Liver·2026
Same author

Invertebrate Automated Phenotyping Platform (INVAPP): An Automated High-Throughput System with Applications in Understanding and Combating Human Diseases.

Methods in molecular biology (Clifton, N.J.)·2026
Same author

The mechanism of pathogenic α<sub>1</sub>-antitrypsin aggregation in the human liver.

Proceedings of the National Academy of Sciences of the United States of America·2025

Related Experiment Video

Updated: Feb 15, 2026

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
08:32

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

4.4K

Stem cell-based therapy for α₁-antitrypsin deficiency.

S Tamir Rashid1, David A Lomas

  • 1Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust/MRC Building, Hills Road, Cambridge CB2 0XY, UK.

Stem Cell Research & Therapy
|February 21, 2012
PubMed
Summary

Human induced pluripotent stem cells can be generated in unlimited quantities for personalized transplants. This study demonstrates their potential for treating genetic disorders by correcting the Z-allele alpha-1-antitrypsin deficiency.

More Related Videos

A Simple Mechanical Procedure to Create Limbal Stem Cell Deficiency in Mouse
04:55

A Simple Mechanical Procedure to Create Limbal Stem Cell Deficiency in Mouse

Published on: November 17, 2016

8.8K
Using the BLT Humanized Mouse as a Stem Cell based Gene Therapy Tumor Model
06:59

Using the BLT Humanized Mouse as a Stem Cell based Gene Therapy Tumor Model

Published on: December 18, 2012

19.3K

Related Experiment Videos

Last Updated: Feb 15, 2026

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
08:32

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

4.4K
A Simple Mechanical Procedure to Create Limbal Stem Cell Deficiency in Mouse
04:55

A Simple Mechanical Procedure to Create Limbal Stem Cell Deficiency in Mouse

Published on: November 17, 2016

8.8K
Using the BLT Humanized Mouse as a Stem Cell based Gene Therapy Tumor Model
06:59

Using the BLT Humanized Mouse as a Stem Cell based Gene Therapy Tumor Model

Published on: December 18, 2012

19.3K

Area of Science:

  • Regenerative Medicine
  • Stem Cell Biology
  • Genetic Medicine

Background:

  • Human induced pluripotent stem cells (hiPSCs) offer a potential source of unlimited cells for autologous transplantation.
  • Inherited genetic disorders represent a significant unmet medical need for novel therapeutic strategies.

Purpose of the Study:

  • To provide proof of principle for using hiPSCs to treat inherited genetic disorders.
  • To demonstrate the correction of the genetic defect in Z-allele alpha-1-antitrypsin deficiency using hiPSCs.

Main Methods:

  • Generation and genetic correction of hiPSCs from patients with alpha-1-antitrypsin deficiency.
  • In vitro characterization of corrected hiPSCs.
  • Assessment of therapeutic potential for alpha-1-antitrypsin deficiency.

Main Results:

  • Successful correction of the genetic defect underlying Z-allele alpha-1-antitrypsin deficiency in hiPSCs.
  • Demonstration of the feasibility of using genetically corrected hiPSCs for potential autologous transplantation.
  • Establishment of a proof-of-concept for hiPSC-based therapy in inherited genetic disorders.

Conclusions:

  • Human induced pluripotent stem cells hold promise for treating inherited genetic disorders.
  • Correction of genetic defects in hiPSCs is achievable.
  • Further research is needed to address safety concerns for clinical translation.