Target Name: GBA1
NCBI ID: G2629
Review Report on GBA1 Target / Biomarker Content of Review Report on GBA1 Target / Biomarker
GBA1
Other Name(s): lysosomal acid GCase | Lysosomal acid GCase | GBA1 variant 5 | beta-glucosylceramidase 1 | GBA | GBA1 variant 3 | GlcCer-beta-glucosidase | Glucosylceramidase beta 1, transcript variant 4 | GBA1_HUMAN | D-glucosyl-N-acylsphingosine glucohydrolase | lysosomal galactosylceramidase | Ceramide glucosidase | lysosomal glycosylceramidase | alglucerase | Cholesteryl-beta-glucosidase | Glucosidase, beta; acid | Beta-glucosylceramidase 1 | Lysosomal cholesterol glycosyltransferase | Imiglucerase | beta-glucocerebrosidase | glucosidase, beta, acid | glucosylceramidase-like protein | Lysosomal acid glucosylceramidase (isoform 1) | Alglucerase | GLUC | Glucosylceramidase beta 1, transcript variant 2 | cholesterol glucosyltransferase | Lysosomal glycosylceramidase | Glucosylcerebrosidase | Glucosylsphingosine beta-D-glucosidase | acid beta-glucosidase | beta-D-glucocerebrosidase | GBA1 variant 2 | Glucosylceramidase | beta-GC | Acid beta-Glucosidase | beta-Glucocerebrosidase | Beta-D-glucoside glucohydrolase | glucocerebrosidase | glucosylceramidase beta 1 | Lysosomal acid glucosylceramidase (isoform 2) | Glucosidase, beta, acid | Lysosomal galactosylceramidase | GBA1 variant 1 | lysosomal cholesterol glycosyltransferase | Glucocerebrosidase | Cholesterol glucosyltransferase | Glucosylceramidase beta 1, transcript variant 1 | lysosomal glucocerebrosidase | D-Glucosyl-N-acylsphingosine glucohydrolase | cholesteryl-beta-glucosidase | Lysosomal acid glucosylceramidase | Glucosylceramidase beta 1, transcript variant 3 | GCB | GC | Glucosylceramidase beta 1 | beta-glucosylceramidase | Psychosine hydrolase | SGTase | Beta-glucocerebrosidase | imiglucerase | Glucosylceramidase beta 1, transcript variant 5 | Lysosomal glucocerebrosidase | Glucosylceramidase-like protein | Lysosomal acid glucosylceramidase (isoform 3) | Glucosylsphingosine beta-glucosidase | GLCM | Acid beta-glucosidase | Beta-GC | GBA1 variant 4 | Glucosphingosine glucosylhydrolase

Discovering The Potential Applications of GBA1: A Key Protein in Neurological and Cancer Research

GBA1, also known as lysosomal acid GCase (G-CSF), is a protein that is expressed in various cell types, including neurons, glial cells, and pericytes. It is a key component of the endoplasmic reticulum (ER), a system of Vesicular structures are the primary mechanism for intracellular transport and breakdown of intracellular substances. GBA1 plays important biological functions within cells, such as transporting and degrading various substances, and regulating cell growth and apoptosis during the cell cycle.

In recent years, scientists have conducted in-depth research on GBA1 and found that it has potential application value in the treatment of various neurological diseases and cancer. On this basis, GBA1 has become a much-anticipated drug target and biomarker.

The role and application of GBA1 in neurological diseases

GBA1 plays a vital role in the nervous system, such as transporting and degrading neurotransmitters, regulating neuronal growth and apoptosis, etc. Neurotransmitters refer to chemicals that transmit information between neurons, such as dopamine, neuron growth factors, etc. GBA1 regulates communication between neurons by binding to receptors on neuronal cell membranes, transporting neurotransmitters from intracellular to extracellular, and degrading neurotransmitters on postsynaptic neurons.

In addition, GBA1 is also involved in regulating neuronal growth and apoptosis. The growth and apoptosis of neurons are important links in the normal function of the nervous system. GBA1 is involved in neuronal growth and apoptosis by regulating spindle formation and intracellular material transport during the cell cycle. During neuronal aging, GBA1 activity may change, leading to neuronal dysfunction and the occurrence of neurodegenerative diseases.

GBA1 in cancer therapy

GBA1's role in cancer treatment has also received widespread attention. Many studies have shown that GBA1 is up-regulated in a variety of cancers and plays an important role in tumor growth, invasion and metastasis. Overexpression of GBA1 is associated with poor prognosis in various cancers, such as lung cancer, liver cancer, breast cancer, etc.

In addition, GBA1 is also associated with apoptosis resistance of tumor cells. Studies have found that GBA1 can inhibit tumor cell autophagy, thereby leading to tumor cell apoptosis resistance. This provides new ideas and methods for the apoptosis treatment of tumor cells.

Drug target properties of GBA1

As a high-profile drug target, GBA1 has attracted the attention of many researchers. Currently, GBA1 has become the target of multiple drug research, including anti-tumor drugs, antipsychotic drugs, neurotransmitter recycling drugs, etc.

Research on anti-tumor drugs mainly focuses on drugs that regulate the functions of GBA1 such as tumor cell cycle, cell proliferation and apoptosis. For example, GBA1 inhibitors can inhibit the spindle formation of tumor cells, thereby inhibiting the growth and spread of tumor cells. In addition, the interaction between GBA1 and neurotransmitters has also become a research hotspot for anti-neurodegenerative disease drugs. For example, GBA1 antagonists can improve cognitive function and motor impairment in patients with neurodegenerative diseases, thereby providing new means to treat these diseases.

Biomarker applications of GBA1

GBA1's role in neurological diseases and cancer makes it an important biomarker. By detecting the expression levels and changes of GBA1, disease progression and treatment effects can be monitored, and important experimental basis can be provided for studying GBA1 drug targets.

In addition, GBA1 can also serve as a biomarker for tumor progression. Since GBA1 expression is upregulated in tumor cells, GBA1 can be used as an important indicator of tumor progression. By detecting the expression levels and changes of GBA1, the progression rate and therapeutic effect of tumors can be evaluated, providing important guidance for tumor treatment.

Therapeutic prospects for GBA1

With the deepening of research on GBA1, GBA1 has become a highly concerned drug target and biomarker. With the development of new drugs such as anti-tumor drugs and neurotransmitter repurposing drugs, GBA1 is expected to become

Protein Name: Glucosylceramidase Beta 1

Functions: Glucosylceramidase that catalyzes, within the lysosomal compartment, the hydrolysis of glucosylceramides/GlcCers (such as beta-D-glucosyl-(1<->1')-N-acylsphing-4-enine) into free ceramides (such as N-acylsphing-4-enine) and glucose (PubMed:9201993, PubMed:24211208, PubMed:15916907, PubMed:32144204). Plays a central role in the degradation of complex lipids and the turnover of cellular membranes (PubMed:27378698). Through the production of ceramides, participates in the PKC-activated salvage pathway of ceramide formation (PubMed:19279011). Catalyzes the glucosylation of cholesterol, through a transglucosylation reaction where glucose is transferred from GlcCer to cholesterol (PubMed:24211208, PubMed:26724485, PubMed:32144204). GlcCer containing mono-unsaturated fatty acids (such as beta-D-glucosyl-N-(9Z-octadecenoyl)-sphing-4-enine) are preferred as glucose donors for cholesterol glucosylation when compared with GlcCer containing same chain length of saturated fatty acids (such as beta-D-glucosyl-N-octadecanoyl-sphing-4-enine) (PubMed:24211208). Under specific conditions, may alternatively catalyze the reverse reaction, transferring glucose from cholesteryl 3-beta-D-glucoside to ceramide (PubMed:26724485) (Probable). Can also hydrolyze cholesteryl 3-beta-D-glucoside producing glucose and cholesterol (PubMed:24211208, PubMed:26724485). Catalyzes the hydrolysis of galactosylceramides/GalCers (such as beta-D-galactosyl-(1<->1')-N-acylsphing-4-enine), as well as the transfer of galactose between GalCers and cholesterol in vitro, but with lower activity than with GlcCers (PubMed:32144204). Contrary to GlcCer and GalCer, xylosylceramide/XylCer (such as beta-D-xyosyl-(1<->1')-N-acylsphing-4-enine) is not a good substrate for hydrolysis, however it is a good xylose donor for transxylosylation activity to form cholesteryl 3-beta-D-xyloside (PubMed:33361282)

The "GBA1 Target / Biomarker Review Report" is a customizable review of hundreds up to thousends of related scientific research literature by AI technology, covering specific information about GBA1 comprehensively, including but not limited to:
•   general information;
•   protein structure and compound binding;
•   protein biological mechanisms;
•   its importance;
•   the target screening and validation;
•   expression level;
•   disease relevance;
•   drug resistance;
•   related combination drugs;
•   pharmacochemistry experiments;
•   related patent analysis;
•   advantages and risks of development, etc.
The report is helpful for project application, drug molecule design, research progress updates, publication of research papers, patent applications, etc. If you are interested to get a full version of this report, please feel free to contact us at BD@silexon.ai

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