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34、很困難。只有少數(shù)性染色體病，可改善患者的第二特征。,二、遺傳病療效的長期評估,遺傳病的治療效果需要有一個謹慎而長期的評價遺傳病治療的初期效果明顯，但長期觀察則達不到預期的目的。例如：女性半乳糖血癥患者在早期的“成功”治療后，到青春期則發(fā)現(xiàn)半乳糖毒性作用導致其卵巢功能早已喪失。,,一些遺傳病的短期治療是有效的，長期治療則會產(chǎn)生一些不良反應。例如：珠蛋白生成障礙性貧血患者經(jīng)輸血治療后會使患者鐵過量； 例如：用凝血因子治療血

35、友病時，患者會因此產(chǎn)生針對輸入的凝血因子的抗體。,,眾多多基因遺傳病的治療應重視環(huán)境條件在多基因病的治療中既要考慮遺傳條件，也要考慮到環(huán)境條件；而在目前狀態(tài)下，環(huán)境條件的改善是多基因遺傳病治療中更為重要的一部分。例如：哮喘病人，過敏病人對過敏原的去除； 高血壓病患者； 糖尿病患者對飲食的控制。,三、雜合子和癥狀前患者的治療,對尚未出現(xiàn)臨床表現(xiàn)的雜合子，癥狀前患者是否應該實施預防性的治療

36、措施不能一概而論，取決于：疾病的嚴重程度治療的近期、遠期效果藥物不良反應大小人們對這種問題的道德取向,四、遺傳病治療的策略,針對突變基因的體細胞基因的修飾與改善；針對突變基因轉(zhuǎn)錄的基因表達調(diào)控；蛋白質(zhì)功能的改善；在代謝水平上對代謝底物或產(chǎn)物的控制；臨床水平的內(nèi)、外科治療以及心理治療等。,傳統(tǒng)的遺傳病的治療方法,一、手術(shù)治療手術(shù)矯正例如：修補和縫合唇裂、腭裂，矯正先天性心臟畸形及兩性畸形等。 器官和組織移植例如

37、：對重型β地中海貧血患者施行骨髓移植術(shù)。,二、藥物治療,去其所余 對于一些因酶促反應障礙，導致體內(nèi)貯積過多的代謝產(chǎn)物，可使用各種理化方法將過多的毒物排除或抑制其生成，使患者的癥狀得到明顯的改善。 應用螯合劑例如：肝豆狀核變性（銅代謝障礙性疾?。?，應用青霉胺與銅離子能形成螯合物的原理，給患者服用青霉胺，可除去患者體內(nèi)細胞中堆積的銅離子。,,應用促排泄劑例如：對于家族性高膽固醇血癥患者可口服消膽胺治療。消膽胺可結(jié)合腸

38、道中的膽酸排出體外，并促使膽固醇更多地轉(zhuǎn)化為膽酸排出體外，降低患者血中膽固醇水平。,,利用代謝抑制劑例如：用別嘌呤醇抑制黃嘌呤氧化酶，可減少體內(nèi)尿酸的形成，可用于治療原發(fā)性痛風和自毀容貌綜合癥。血漿置換或血漿過濾（除去大量含有毒物的血液） 　例如：應用于重型高膽固醇血癥的治療。,,平衡清除法 對于某些溶酶體貯積癥，由于其沉積物可彌散入血，并保持血與組織之間的動態(tài)平衡。如果把一定的酶制劑注入血液以清除底

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50、以改善患者的體格發(fā)育。例如：糖尿病患者注射胰島素等均可使癥狀得到明顯的改善。但這種補充常需終生進行才能維持療效。例如：先天性無丙種球蛋白血癥患者，給予丙種球蛋白制劑，。,酶療法 遺傳性代謝病通常是由于基因突變造成酶的缺失或活性降低，可用酶誘導和酶補充的方法進行治療。酶誘導治療例如：雄激素能誘導α1-抗胰蛋白酶的合成，因而可應用于α1-抗胰蛋白酶缺乏癥的治療。酶補充療法例如：嚴重的α1-抗胰蛋白酶缺乏癥患者每周用4g

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66、的基因治療遺傳性疾病疾 病 傳遞的基因或產(chǎn)物 靶細胞或組織 載 體α-抗胰蛋白酶缺乏癥 α-抗胰蛋白酶 呼吸道 脂質(zhì)體慢性肉芽腫 P47PHoX 骨髓細胞

67、 逆轉(zhuǎn)錄病毒囊性纖維化 囊性纖維化跨膜調(diào)節(jié)蛋白 呼吸道 腺病毒/脂質(zhì)體 腺伴隨病毒家族性高膽固

68、醇血癥 低密度脂蛋白受體 肝細胞 逆轉(zhuǎn)錄病毒范可尼綜合征 互補組C基因 造血祖細胞 逆轉(zhuǎn)錄病毒高雪病 葡糖腦苷脂酶 周圍血細胞 逆轉(zhuǎn)錄病毒