Related Experiment Video

Updated: Aug 17, 2025

Author Spotlight: Expression and Purification of Human Solute Carrier Transporters Using Codon-Optimized Genes
07:10

Author Spotlight: Expression and Purification of Human Solute Carrier Transporters Using Codon-Optimized Genes

Published on: September 29, 2023

3.8K

ABC transporters: human disease and pharmacotherapeutic potential

Jonathan M Moore1, Eric L Bell1, Robert O Hughes1

  • 1Rectify Pharmaceuticals, Cambridge, MA, USA.

Trends in Molecular Medicine
|December 12, 2022
PubMed

Insights

Adenosine triphosphate (ATP)-binding cassette (ABC) transporters are crucial membrane proteins involved in human health and disease. This review highlights their role in various disorders and potential as drug targets.

Area of Science:

  • Biochemistry
  • Molecular Biology
  • Human Physiology

Background:

  • Adenosine triphosphate (ATP)-binding cassette (ABC) transporters constitute a large superfamily of membrane proteins.
  • These proteins actively transport diverse substrates across cellular membranes, playing vital roles in human biology.

Purpose of the Study:

  • To explore the significant relevance of ABC transporters in human diseases.
  • To discuss their potential as targets for small-molecule drug discovery.

Main Methods:

  • Literature review of ABC transporter functions.
  • Analysis of ABC transporters' roles in monogenic and complex diseases.
  • Examination of therapeutic strategies targeting ABC transporters.

Main Results:

  • Over 21 ABC transporters are linked to rare genetic disorders.
  • Numerous ABC transporters are implicated in common and complex diseases.
  • CFTR modulator therapies demonstrate the therapeutic potential of targeting ABC transporters.

Conclusions:

  • ABC transporters are central to human (patho)physiology.
  • Targeting ABC transporters offers promising avenues for drug development.
  • Further research into ABC transporters can lead to novel therapeutic interventions.
Keywords:
ABC transporterABCA7ATP-binding cassetteCFTR/ABCC7genetic diseasepharmacotherapies

More Related Videos

Setting-up an In Vitro Model of Rat Blood-brain Barrier BBB: A Focus on BBB Impermeability and Receptor-mediated Transport16:26

Setting-up an In Vitro Model of Rat Blood-brain Barrier BBB: A Focus on BBB Impermeability and Receptor-mediated Transport

Published on: June 28, 2014

42.6K
Using Multi-fluorinated Bile Acids and In Vivo Magnetic Resonance Imaging to Measure Bile Acid Transport08:42

Using Multi-fluorinated Bile Acids and In Vivo Magnetic Resonance Imaging to Measure Bile Acid Transport

Published on: November 27, 2016

11.3K

Related Concept Videos

ABC Transporters: Exporter01:31

ABC Transporters: Exporter

ATP-binding cassette or ABC transporter is the largest superfamily of integral membrane proteins. The transporters have transmembrane-binding domains (TMDs) and nucleotide-binding domains (NBDs). The TMDs are specific to their substrates, whereas the NBDs are similar to engines that complete ATP hydrolysis to complete the substrate transport. They can be full transporters consisting of two TMDs and NBDs, half transporters with one TMD and NBD, while some encoded with a single TMD or NBD are...
4.5K
ABC Transporters: Importer01:27

ABC Transporters: Importer

ATP-binding cassette or ABC transporters are a class of ATP-driven pumps that hydrolyze ATP to move solutes across the membrane. They can be grouped into importers and exporters. While exporters are present in all domains of life, importers exist only in bacteria and some plants.
In bacteria, based on the number of transmembrane helices and the chemical nature of their substrates, the ABC importers can be divided into three types:
2.8K
Carrier-Mediated Transport01:06

Carrier-Mediated Transport

Carrier-mediated transport is a pivotal process in drug absorption, particularly for lipid-insoluble drugs, and encompasses facilitated diffusion and active transport. Facilitated diffusion allows drugs to move along their concentration gradient without energy expenditure, while active transport utilizes ATP to drive drug movement against this gradient.
Active transport involves two types of membrane-spanning transporters: uptake and efflux. Uptake transporters are expressed in the small...
491
Active Transport01:14

Active Transport

Active transport is a critical biological process that allows cells to move solutes against an electrochemical gradient. This process requires direct energy input and is characterized by its selectivity, saturability, and susceptibility to competitive inhibition.
Primary active transporters, like Na+, K+ and -ATPase, directly utilize ATP to move ions across the membrane. These transporters play significant roles in various physiological processes. For instance, Na+, K+ and -ATPase maintain...
796
Membrane Transporters01:31

Membrane Transporters

Transporters are essential membrane transport proteins with functions related to cell nutrition, homeostasis, communication, etc. Approximately 7% of all genes in the human genome code for transporters or transporter-related proteins.
Transporters are mainly composed of alpha-helices, built from bundles of ten or more helices traversing the plasma membrane. The solute-binding sites are located midway, where some of the helices are broken or distorted, making space for the binding site through...
12.0K
Hepatic Drug Clearance: Role of Transporters01:14

Hepatic Drug Clearance: Role of Transporters

In the liver and bile canaliculi, influx and efflux transporters modification can influence intrinsic clearance. Transporters play a significant role in moving drugs within liver cells. Elaborate models, such as the Biopharmaceutical Classification System (BCS), are essential to relate transporters to drug disposition. This system categorizes drugs into four classes based on solubility and permeability, providing insights into elimination routes and the effects of transporters following oral...
93