Abstract: The present application relates to a gRNA targeting polypeptide 2, subfamily V, family 4, cytochrome P450 (CYP4V2) gene. The present application also relates to a donor nucleic acid molecule comprising a human CYP4V2 nucleotide sequence. The present application further relates to use of the gRNA and the donor nucleic acid molecule in the treatment of Bietti crystalline dystrophy.

Abstract: The present application relates to expression cassette combinations and uses thereof. The expression cassette combination comprises a first expression cassette and a second expression cassette, wherein the first expression cassette can express an N-terminal truncated version of an ABCA4 protein, the second expression cassette can express a C-terminal truncated version of an ABCA4 protein, and the N-terminal truncated version of the ABCA4 protein and the C-terminal truncated version of the ABCA4 protein can possess the biological functions of a complete ABCA4 protein without forming the complete ABCA4 protein therefrom.

Abstract: The present application provides a PRPF31 variant comprising an exogenous nuclear localization (NLS) domain, wherein the exogenous NLS domain comprises an amino acid sequence set forth in any of SEQ ID NOs. 3 to 4. The PRPF31 variant can improve the biological activity of a retinal pigment epithelium (RPE) cell, and can treat a disease caused by PRPF31 gene mutation.

Abstract: The present application relates to a vector comprising a polynucleotide encoding CYP4V2 and a promoter operably linked to the polynucleotide encoding CYP4V2. The vector described herein has a good expression effect, high expression speed, and more stable expression intensity. The present application also provides cells, pharmaceutical compositions, and kits comprising the vector, and methods for treating, alleviating, and/or preventing a disease or disorder associated with retinal pigment epithelium (RPE) atrophy.

Abstract: The present application relates to a gRNA targeting CYP4V2 gene, and a donor nucleic acid molecule comprising a CYP4V2 gene fragment. The present application also relates to use of the gRNA and the donor nucleic acid molecule in the preparation of a medicament for treating Bietti crystalline dystrophy.

Abstract: The present application relates to the use of CYP4V2 and RdCVF in the manufacture of a medicament for treating, alleviating, and/or preventing a disease or disorder associated with retinal pigment epithelium (RPE) atrophy. The present application also relates to an isolated nucleic acid molecule comprising a polynucleotide encoding CYP4V2 and a polynucleotide encoding RdCVF. The present application also relates to an amino acid sequence encoded by the isolated nucleic acid molecule, a vector comprising the isolated nucleic acid molecule, and a cell comprising the nucleic acid or the vector as well as use thereof in the manufacture of a medicament for treating, alleviating, and/or preventing a disease or disorder associated with retinal pigment epithelium (RPE) atrophy.

Abstract: A vector includes a polynucleotide encoding CYP4V2 and a promoter operably linked to the polynucleotide encoding CYP4V2. The vector has a good expression effect, high expression speed, and more stable expression intensity. Cells, pharmaceutical compositions, and kits can include the vector. Methods for treating, alleviating, and/or preventing a disease or disorder associated with retinal pigment epithelium (RPE) atrophy can include administering the vector to a subject.

Abstract: The present application relates to a fusion protein and use thereof. The fusion protein comprises RdCVF and a signal peptide, wherein the signal peptide is an amino acid sequence set forth in any one of SEQ ID NOs: 27-31 and 33. The present application also relates to an isolated nucleic acid molecule comprising a) a polynucleotide encoding RdCVF and b) a polynucleotide encoding a signal peptide. The present application also provides a vector, a cell, and a pharmaceutical composition containing the nucleic acid molecule, as well as medical use thereof. The fusion protein and/or isolated nucleic acid molecule described herein can effectively increase the secretion of RdCVF.

Abstract: The present application relates to the use of CYP4V2 and RdCVF in the manufacture of a medicament for treating, alleviating, and/or preventing a disease or disorder associated with retinal pigment epithelium (RPE) atrophy. The present application also relates to an isolated nucleic acid molecule comprising a polynucleotide encoding CYP4V2 and a polynucleotide encoding RdCVF. The present application also relates to an amino acid sequence encoded by the isolated nucleic acid molecule, a vector comprising the isolated nucleic acid molecule, and a cell comprising the nucleic acid or the vector as well as use thereof in the manufacture of a medicament for treating, alleviating, and/or preventing a disease or disorder associated with retinal pigment epithelium (RPE) atrophy.

Abstract: The present invention relates to herbal compositions and herbal extracts useful for treating cerebral vascular disease, including stroke, intracranial hemorrhage, intracranial infarction, and vascular dementia. The present invention can be used to reduce mortality rate and improve the quality of life of an individual after pathological injury that results in cerebrovascular and/or neuronal disorders. Further, the invention relates to the treatment of acute intracerebral hemorrhage by the novel herbal formulation PHY828 and provides methods of making PHY828 compositions.

Abstract: An adaptive control artificial wavemaking device comprises an air blower as shock wave source. According to the invention, the device further comprises a control system consisted of a float, a sensor, a control circuit and electromagnetic actuators; butterfly valves; and air chamber for generating shock wave. When the sensor receives signals from the flaot, the signals are transferred through the control circuit to actuate the electromagnetic actuators to control opening and closing of said butterfly valves to enable the air chamber to generate a shock wave which is in resonance with the water wave. The device may further comprises an oscillator for generating shock wave of a given frequence during starting. The device according to the invention has the advantage of similified structure, low mangufacture cost and low energy consumption, thus it may be widely used for aquatic breeding, sport, recreation and medical facilities.