ML-SA1 is a chemical compound which acts as an "agonist" (i.e. channel opener) of the

TRPML TRPML (transient receptor potential cation channel, mucolipin subfamily) comprises a group of three evolutionarily related proteins that belongs to the large family of transient receptor potential ion channels. The three proteins TRPML1, TRPML2 ...

family of calcium channels. It has mainly been studied for its role in activating

TRPML1 Mucolipin-1 also known as TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) is a protein that in humans is encoded by the ''MCOLN1'' gene. It is a member of the small family of the TRPML channels, a subgroup of ...

channels, although it also shows activity at the less studied

TRPML2 Mucolipin-2 also known as TRPML2 (transient receptor potential cation channel, mucolipin subfamily, member 2) is a protein that in humans is encoded by the ''MCOLN2'' gene In biology, the word gene (from , ; "... Wilhelm Johannsen coined th ...

and

TRPML3 Mucolipin-3 also known as TRPML3 (transient receptor potential cation channel, mucolipin subfamily, member 3) is a protein that in humans is encoded by the ''MCOLN3'' gene. It is a member of the small family of the TRPML channels, a subgroup o ...

subtypes. TRPML1 is important for the function of

lysosome A lysosome () is a membrane-bound organelle found in many animal cells. They are spherical vesicles that contain hydrolytic enzymes that can break down many kinds of biomolecules. A lysosome has a specific composition, of both its membrane ...

s, and ML-SA1 has been used to study several disorders resulting from impaired lysosome function, including

mucolipidosis type IV Mucolipidosis type IV (ML IV, ganglioside sialidase deficiency, or ML4) is an autosomal recessive lysosomal storage disorder. Individuals with the disorder have many symptoms including delayed psychomotor development and various ocular aberratio ...

and Niemann-Pick's disease type C, as well as other conditions such as stroke and Alzheimer's disease.

References

{{Transient receptor potential channel modulators Phthalimides Amides