Details of the Drug Therapeutic Target (DTT)

General Information of Drug Therapeutic Target (DTT) (ID: TTULDG7)

• DTT Name: Lysosome-associated membrane glycoprotein 2 (LAMP2)
• Synonyms: LAMP-2; Lysosome-associated membrane protein 2; CD107 antigen-like family member B; LGP-96; DE AltName: CD_antigen=CD107b
• Gene Name: LAMP2
• DTT Type: Clinical trial target; [1]
• UniProt ID: LAMP2_HUMAN
• TTD ID: T46052
• Sequence:
  MVCFRLFPVPGSGLVLVCLVLGAVRSYALELNLTDSENATCLYAKWQMNFTVRYETTNKT
  YKTVTISDHGTVTYNGSICGDDQNGPKIAVQFGPGFSWIANFTKAASTYSIDSVSFSYNT
  GDNTTFPDAEDKGILTVDELLAIRIPLNDLFRCNSLSTLEKNDVVQHYWDVLVQAFVQNG
  TVSTNEFLCDKDKTSTVAPTIHTTVPSPTTTPTPKEKPEAGTYSVNNGNDTCLLATMGLQ
  LNITQDKVASVININPNTTHSTGSCRSHTALLRLNSSTIKYLDFVFAVKNENRFYLKEVN
  ISMYLVNGSVFSIANNNLSYWDAPLGSSYMCNKEQTVSVSGAFQINTFDLRVQPFNVTQG
  KYSTAQDCSADDDNFLVPIAVGAALAGVLILVLLAYFIGLKHHHAGYEQF
• Function: Plays an important role in chaperone-mediated autophagy, a process that mediates lysosomal degradation of proteins in response to various stresses and as part of the normal turnover of proteins with a long biological half-live. Functions by binding target proteins, such as GAPDH and MLLT11, and targeting them for lysosomal degradation. Plays a role in lysosomal protein degradation in response to starvation (By similarity). Required for the fusion of autophagosomes with lysosomes during autophagy. Cells that lack LAMP2 express normal levels of VAMP8, but fail to accumulate STX17 on autophagosomes, which is the most likely explanation for the lack of fusion between autophagosomes and lysosomes. Required for normal degradation of the contents of autophagosomes. Required for efficient MHCII-mediated presentation of exogenous antigens via its function in lysosomal protein degradation; antigenic peptides generated by proteases in the endosomal/lysosomal compartment are captured by nascent MHCII subunits. Is not required for efficient MHCII-mediated presentation of endogenous antigens.
• KEGG Pathway:
  Autophagy - animal (hsa04140)
  Lysosome (hsa04142)
  Phagosome (hsa04145)
  Tuberculosis (hsa05152)
• Reactome Pathway:
  Neutrophil degranulation (R-HSA-6798695)
  Chaperone Mediated Autophagy (R-HSA-9613829)
  Platelet degranulation (R-HSA-114608)

Molecular Interaction Atlas (MIA) of This DTT

1 Clinical Trial Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
RP-A501	DMHZ24N	Type 2 glycogen storage disease	5C51.3	Phase 1	[1]

References

• [1] ClinicalTrials.gov (NCT03882437) Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B. U.S. National Institutes of Health.